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| bluebird sickle cell gene therapy: Exploring Novel Clinical Trial Designs for Gene-Based Therapies National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Forum on Regenerative Medicine, 2020-08-27 Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop. |
| bluebird sickle cell gene therapy: Sickle Cell Pain Samir K. Ballas, 2015-06-01 Sickle Cell Pain is a panoramic, in-depth exploration of every scientific, human, and social dimension of this cruel disease. This comprehensive, definitive work is unique in that it is the only book devoted to sickle cell pain, as opposed to general aspects of the disease. The 752-page book links sickle cell pain to basic, clinical, and translational research, addressing various aspects of sickle pain from molecular biology to the psychosocial aspects of the disease. Supplemented with patient narratives, case studies, and visual art, Sickle Cell Pain’s scientific rigor extends through its discussion of analgesic pharmacology, including abuse-deterrent formulations. The book also addresses in great detail inequities in access to care, stereotyping and stigmatization of patients, the implications of rapidly evolving models of care, and recent legislation and litigation and their consequences. |
| bluebird sickle cell gene therapy: Building Breakthroughs Raju Prasad, 2022-12-06 The author tells stories about medical breakthroughs. The stories are based on in-depth interviews with both patients who have benefited from the breakthroughs and the scientists who were responsible for them-- |
| bluebird sickle cell gene therapy: The Song of the Cell Siddhartha Mukherjee, 2022-10-30 From Pulitzer Prize-winning and #1 New York Times bestselling author of The Emperor of All Maladies and The Gene, The Song of The Cell is the third book in this extraordinary writer's exploration of what it means to be human-rich with Siddhartha Mukherjee's revelatory and exhilarating stories of scientists, doctors, and all the patients whose lives may be saved by their work. In the late 1600s, a distinguished English polymath, Robert Hooke, and an eccentric Dutch cloth merchant, Antonie van Leeuwenhoek, look down their handmade microscopes. What they see introduces a radical concept that sweeps through biology and medicine, touching virtually every aspect of the two sciences and altering both forever. It is the fact that complex living organisms are assemblages of tiny, self-contained, self-regulating units. Our organs, our physiology, our selves-hearts, blood, brains-are built from these compartments. Hooke christens them 'cells'. The discovery of cells-and the reframing of the human body as a cellular ecosystem-announced the birth of a new kind of medicine based on the therapeutic manipulations of cells. A hip fracture, a cardiac arrest, Alzheimer's, dementia, AIDS, pneumonia, lung cancer, kidney failure, arthritis, COVID-all could be viewed as the results of cells, or systems of cells, functioning abnormally. And all could be perceived as loci of cellular therapies. In The Song of the Cell, Mukherjee tells the story of how scientists discovered cells, began to understand them, and are now using that knowledge to create new humans. He seduces readers with writing so vivid, lucid, and suspenseful that complex science becomes thrilling. Told in six parts, laced with Mukherjee's own experience as a researcher, doctor, and prolific reader, The Song of the Cell is both panoramic and intimate-a masterpiece. |
| bluebird sickle cell gene therapy: Gene and Cell Therapies for Beta-Globinopathies Punam Malik, John Tisdale, 2017-11-09 Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipulation of the defective organ might be equally therapeutic. However, procedural toxicities and the requirement of an HLA-matched sibling donor limit this approach to a fraction of affected individuals. The editors review the progress & the state of the field in HSCT for hemoglobinopathies & shed light on the major changes expected in the next decade. Although allogeneic HSCT is a curative option, it is limited by the availability of matched donors, which are often available only to 15-20% of patients. An alternative to allogeneic HS CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally, targeted genetic correction is emerging as a novel therapeutic strategy in the hemoglobinopathies. Although ideal, the inefficiency of targeted correction was rate limiting for translation of this technology to the clinic. With advancements in zinc finger nucleases and TALE endonuclease mediated targeted correction, correction frequencies in hematopoietic stem cells is now reaching levels that may become clinically relevant. Furthermore, the ability to generate autologous embryonic stem cell like cells from primary somatic cells (skin fibroblasts or hematopoietic cells) of the affected individual has allowed for the potential application of genetic correction strategies.This Book reviews upcoming genetic strategies to reactivate fetal hemoglobin production and research advances. |
| bluebird sickle cell gene therapy: Gene Therapy, An Issue of Hematology/Oncology Clinics of North America Daniel E. Bauer, Donald B Kohn, 2017-09-27 This issue of Hematology/Oncology Clinics will focus on Gene Therapy. Topics include, but are not limited to Historical Perspective and Current Renaissance, Integrating Vectors, Nonintegrating Vectors, Gene Editing, Conditioning Therapies for Autologous HSCT, Approaches to Immunodeficiency, Approaches to Hemoglobinopathy, Approaches to Hemophilia, Hematopoietic Gene Therapies for Neurologic and Metabolic Disease, Gene Therapy Approaches to HIV and other Infectious Diseases, HSC Approaches to Cancer, and Gene Modified T Cell Therapies for Cancer. |
| bluebird sickle cell gene therapy: Second Generation Cell and Gene-Based Therapies Alain Vertes, Nathan J. Dowden, Devyn Smith, Nasib Qureshi, 2020-02-07 Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products.The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. - Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization - Perspectives section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights - Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies |
| bluebird sickle cell gene therapy: Pediatric Clinics , 2008 |
| bluebird sickle cell gene therapy: Disorders of Hemoglobin Martin H. Steinberg, 2009-08-17 Completely revised new edition of the definitive reference on disorders of hemoglobin. |
| bluebird sickle cell gene therapy: The Business of Healthcare Innovation Lawton Robert Burns, 2005-08-25 The Business of Healthcare Innovation is the first wide-ranging analysis of business trends in the manufacturing segment of the health care industry. In this leading edge volume, Professor Burns focuses on the key role of the 'producers' as the main source of innovation in health systems. Written by professors of the Wharton School and industry executives, this book provides a detailed overview of the pharmaceutical, biotechnology, genomics/proteomics, medical device and information technology sectors. It analyses the market structures of these sectors as well as the business models and corporate strategies of firms operating within them. Most importantly, the book describes the growing convergence between these sectors and the need for executives in one sector to increasingly draw upon trends in the others. It will be essential reading for students and researchers in the field of health management, and of great interest to strategy scholars, industry practitioners and management consultants. |
| bluebird sickle cell gene therapy: The Molecular Basis of Blood Diseases George Stamatoyannopoulos, 2001 The superb Third Edition of this popular text covers all the recent groundbreaking developments which have taken place in this field. Comprehensively revised, it presents all the latest findings on the molecular bases of blood cell functions and disease mechanisms and the impact of these discoveries on the state of medicine. This edition includes new chapters such as signaling and antigen presentation by B-lymphocytes, molecular oncogenesis and more! |
| bluebird sickle cell gene therapy: Renaissance Of Sickle Cell Disease Research In The Genome Era Betty Pace, 2007-01-24 The Human Genome Project has spawned a Renaissance of research faced with the daunting expectation of personalized medicine for individuals with sickle cell disease in the Genome Era. This book offers a comprehensive and timeless account of emerging concepts in clinical and basic science research, and community concerns of health disparity to educate professionals, students and the general public about meeting this challenging expectation. Contributions from physicians, research scientists, scientific administrators and community workers make Renaissance of Sickle Cell Disease Research in the Genome Era unique among the catalogue of books on this genetic disorder.Part 1 offers detailed review of the National Heart Lung and Blood Institute's leadership role in funding sickle cell research, as well as developing progressive research initiatives and the predicted impact of the Human Genome Project. Part 2 gives an account of several clinical research perspectives based on the Cooperative Study of Sickle Cell Disease. These include recommendations for newborn screening, pain management, stroke, transfusion therapy and pediatric and adult healthcare. Part 3 offers novel insights into basic science research progress and the impact of the Human Genome Project on the direction of hemoglobinopathy research, including hemoglobin switching, bone marrow transplantation and gene therapy. Part 4 engages the reader in a culture-based discussion of the stigma attached to sickle cell disease in the African American community and the apprehensions about genetic research in this community. It concludes with a global perspective on sickle cell disease from African, European and American experiences. For readers seeking a definitive account of sickle cell disease appropriate for students, researchers and community workers, this collaborative effort is an ideal textbook./a |
| bluebird sickle cell gene therapy: Hematopoietic Stem Cells Meng Zhao, Pengxu Qian, 2024-01-17 This book renders a comprehensive understanding of hematopoietic stem cells (HSCs) from their embryonic development through adult maintenance to aging, in the studies conducted in zebrafish and mammals. Hematopoiesis provides a paradigm for understanding the development, maintenance, regeneration, aging and malignant transformation of mammalian organs. Sitting at the apex of the hematopoiesis hierarchy tree, HSCs orchestrate their proliferation, self-renewal, and differentiation to produce all the blood cell lineages throughout life, which represents the best example for somatic stem cell studies. In this book, key regulatory mechanisms for HSC self-renewal and differentiation are overviewed in an array of fields including epigenetics, metabolism and microenvironment regulation. It also highlights the HSC heterogeneity and clonal dynamics from the recent advanced single-cell technologies. This book elaborates on the research history of HSC studies and reveals how the insights from HSC studies shed light on their clinic application. It presents great value from the bench to the clinic. |
| bluebird sickle cell gene therapy: Gene and Cell Therapies Eve Hanna, Mondher Toumi, 2020-05-19 The major advances in the field of biotechnology and molecular biology in the twenty-first century have led to a better understanding of the pathophysiology of diseases. A new generation of biopharmaceuticals has emerged, including a wide and heterogeneous range of innovative cell and gene therapies. These therapies aim to prevent or treat chronic and serious life-threatening diseases, previously considered incurable. This book describes the evolution and adaptation of the regulatory environment to assess these therapies in contrast with the resistance of health technology assessment (HTA) agencies and payers to acknowledge the specificity of cell and gene therapies and the need to adapt existing decision-making frameworks. This book provides insights on the learnings from the experience of current cell and gene therapies (regulatory approval, HTA, and market access), in addition to future trends to enhance patient access to these therapies. Key Features: Describes the potential change of treatment paradigm and the specificity of cell and gene therapies, including the gradual move from repeated treatment administration to one-time single administration with the potential to be definite cure Highlights the challenges at the HTA level Discusses the affordability of future cell and gene therapies and the possible challenges for health insurance systems Provides potential solutions to address these challenges and ensure patient access to innovation while maintaining the sustainability of healthcare systems |
| bluebird sickle cell gene therapy: Benign Hematologic Disorders in Children Deepak M. Kamat, Melissa Frei-Jones, 2020-08-18 This book provides a comprehensive overview of benign hematologic disorders in children. Divided into nine sections, the text reviews common hematologic disorders or conditions that affect children, while providing state-of-the-art information on pathophysiology, diagnosis, treatment, and management strategies. The text begins with a section on hematopoiesis, and the next section covers red blood cell disorders. The following sections provide overviews of platelet disorders, white blood cell disorders, and coagulation disorders. The sixth and seventh sections discuss neonatal hematology and bone marrow failure syndrome. The eighth section reviews supportive care, while the final section covers miscellaneous subjects including pediatric vascular anomalies and complement dysregulation syndromes. Written by experts in the field, Benign Hematologic Disorders in Children: A Clinical Guide is a valuable resource for clinicians and practitioners who treat children afflicted with these disorders. |
| bluebird sickle cell gene therapy: The Thalassemias D. J. Weatherall, 1983 |
| bluebird sickle cell gene therapy: Developments in sickle cell disease therapy and potentials for gene therapy Robert W. Maitta, Hollie Marie Reeves, Magali J. Fontaine, France Pirenne, 2023-07-03 |
| bluebird sickle cell gene therapy: Protocols in Advanced Genomics and Allied Techniques Aruna Pal, 2021-11-14 This laboratory manual includes the latest tools and techniques involved in genomic research. It starts with an introductory chapter on genomics and the various tools and applications involved. The initial chapters present protocols for basic techniques such as DNA isolation, electrophoresis, PCR, cDNA synthesis etc. The book then goes on to describe more advanced techniques such as next-generation sequencing, exome sequencing, use of RNAi, RNAseq, genome editing, single cell genomics etc. Each topic includes a brief description, information on the principles involved, materials & methods, protocol, and expected results, with diagrams and graphs. All protocols are presented in a very lucid and precise way, to make it easy for readers to follow and replicate them. |
| bluebird sickle cell gene therapy: The Changing Economics of Medical Technology Institute of Medicine, Committee on Technological Innovation in Medicine, 1991-02-01 Americans praise medical technology for saving lives and improving health. Yet, new technology is often cited as a key factor in skyrocketing medical costs. This volume, second in the Medical Innovation at the Crossroads series, examines how economic incentives for innovation are changing and what that means for the future of health care. Up-to-date with a wide variety of examples and case studies, this book explores how payment, patent, and regulatory policiesâ€as well as the involvement of numerous government agenciesâ€affect the introduction and use of new pharmaceuticals, medical devices, and surgical procedures. The volume also includes detailed comparisons of policies and patterns of technological innovation in Western Europe and Japan. This fact-filled and practical book will be of interest to economists, policymakers, health administrators, health care practitioners, and the concerned public. |
| bluebird sickle cell gene therapy: Prenatal Gene Therapy Charles Coutelle, Simon N. Waddington, 2012-05-31 The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions. In Prenatal Gene Therapy: Concepts, Methods and Protocols, expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These include detailed protocols for vector production, for breeding and husbandry of the animal models, for the surgical procedures of gene delivery in large and small animals and for the methods of gene transfer analysis. Written in the highly successful Methods in Molecular BiologyTM series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls. Thorough and intuitive, Prenatal Gene Therapy: Concepts, Methods and Protocols seeks to aid scientists in the further study of prenatal disease and gene therapy projects beyond the scope of fetal medicine. |
| bluebird sickle cell gene therapy: Mutation-Specific Gene Editing for Blood Disorders Carsten Werner Lederer, Pietro Genovese, Annarita Miccio, Sjaak Philipsen, 2021-11-25 Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com. |
| bluebird sickle cell gene therapy: Sickle Cell Syndromes, An Issue of Hematology/Oncology Clinics of North America, E-Book Sophie Lanzkron, Jane Little, 2022-11-20 In this issue of Hematology/Oncology Clinics, guest editors Drs. Sophie Lanzkron and Jane Little bring their considerable expertise to the topic of Sickle Cell Syndromes. Top experts in the field cover key topics such as structural racism and impact on sickle cell disease (SCD); pathophysiology and biomarkers of SCD; genetic modifiers of SCD; allogeneic transplant and gene therapy: reproductive health; chronic pain; and more. - Contains 16 relevant, practice-oriented topics including innovative therapies, addressing challenging complications, novel science on mechanisms of disease; preventing cognitive decline in people with SCD; quality of life in SCD; and more. - Provides in-depth clinical reviews on sickle cell syndromes, offering actionable insights for clinical practice. - Presents the latest information on this timely, focused topic under the leadership of experienced editors in the field. Authors synthesize and distill the latest research and practice guidelines to create clinically significant, topic-based reviews. |
| bluebird sickle cell gene therapy: Curing Genetic Diseases through Genome Reprogramming , 2021-06-24 Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome. - Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing - Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects - Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations |
| bluebird sickle cell gene therapy: Advances in Application of Stem Cells: From Bench to Clinics Firdos Alam Khan, 2021-09-14 The field of stem cell biology is expanding with a continued surge of new information related to its applications. Over past few years, stem cells have been extensively used in cell therapy, tissue engineering, in vitro drug testing among others. At the moment there is no single book available which comprehensively describes the significance of various application of stem cells derived from embryonic and adult sources from lab to clinics. In this edited volume, we discuss basics and advanced topics of stem cells to help researchers, students and professional find the most important information in a single source of updated information about stem cells and relevant applications. This book is divided in 12 chapters and covers topics such as in vitro cell culture, 3D cell culture, cell therapy, tissue engineering, cell factory, cell functionality, in vitro drug testing, organ development, autologous transplantation, allogeneic transplantation, adult stem cells, multipotent stem cells, induced pluripotent stem cells, a pluripotent and embryonic stem cells. |
| bluebird sickle cell gene therapy: Thalassemia and Other Hemolytic Anemias Isam Jaber Al-Zwaini, 2018-07-11 Thalassemia is a very common disease first described by pediatrician Thomas Benton Cooley in 1925 who described it in a patient of Italian origin. At that time, it was designated as Cooley's anemia. George Hoyt Whipple, a Nobel prize winner, and W. L. Bradford, a professor of pediatrics at the University of Rochester, coined the term thalassemia in 1936, which in Greek means anemia of the sea (Thalassa means sea, and emia means blood), due to the fact that it is very common in the area of the Mediterranean Sea. This name is actually misleading because it can occur everywhere in the world. Thalassemia is not a single disease; it is rather a group of hereditary disorders of the production of globulin chain of the hemoglobin. Throughout the world, thalassemia affects approximately 4.4 of every 10,000 live births. It represents a major social and emotional impact on the patient and his family and a major burden on health services where the prevalence is high. |
| bluebird sickle cell gene therapy: Juvenescence Jim Mellon, Al Chalabi, 2017-09-25 |
| bluebird sickle cell gene therapy: Vogel and Motulsky's Human Genetics Michael Speicher, Stylianos E. Antonarakis, Arno G. Motulsky, 2009-11-26 The fourth edition of this classical reference book can once again be relied upon to present a cohesive and up-to-date exposition of all aspects of human and medical genetics. Human genetics has become one of the main basic sciences in medicine, and molecular genetics is increasingly becoming a major part of this field. This new edition integrates a wealth of new information - mainly describing the influence of the molecular revolution - including the principles of epigenetic processes which together create the phenotype of a human being. Other revisions are an improved layout, sub-division into a larger number of chapters, as well as two-colour print throughout for ease of reference, and many of the figures are now in full colour. For graduates and those already working in medical genetics. |
| bluebird sickle cell gene therapy: Discrete Event Simulation for Health Technology Assessment J. Jaime Caro, Jörgen Möller, Jonathan Karnon, James Stahl, Jack Ishak, 2015-10-16 This is the first book to make all the central concepts of discrete event simulation relevant for health technology assessment. Accessible to beginners, the book requires no prerequisites and describes the concepts with as little jargon as possible. It presents essential concepts, a fully worked out implementation example, approaches to analyze the simulations, the development of the required equations, model verification techniques, and validation. The book also covers various special topics and includes a real case study involving screening strategies for breast cancer surveillance. |
| bluebird sickle cell gene therapy: Evidence-Based Management of Sickle Cell Disease M D George R Buchanan, M D M P H Araba N Afenyi-Annan, M D Samir K Ballas, 2014-09-09 Sickle cell disease can be severe and disabling. When properly treated, patients live longer and with better quality life. This is a US government publication intended to provide evidence-based guidelines for the care of these patients for the use of all concerned providers as well as patients and family members. This book is available in print here for convenience. |
| bluebird sickle cell gene therapy: The EBMT Handbook Nicolaus Kröger, Mohamad Mohty, Carlo Dufour, 2020-10-08 This Open Access edition of the European Society for Blood and Marrow Transplantation (EBMT) handbook addresses the latest developments and innovations in hematopoietic stem cell transplantation and cellular therapy. Consisting of 93 chapters, it has been written by 175 leading experts in the field. Discussing all types of stem cell and bone marrow transplantation, including haplo-identical stem cell and cord blood transplantation, it also covers the indications for transplantation, the management of early and late complications as well as the new and rapidly evolving field of cellular therapies. This book provides an unparalleled description of current practices to enhance readers' knowledge and practice skills. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors. |
| bluebird sickle cell gene therapy: Life’s Blood Michael H. Rosove, |
| bluebird sickle cell gene therapy: Pharma and Profits John L. LaMattina, 2022-07-06 High-level commentary on various facets of the pharmaceutical industry from a key leader in the field This book clearly explains the value that the pharmaceutical industry offers to society which is often underreported against the more negative topic of high drug prices. It also offers an overview for drug discovery and development professionals, highlighting the challenges that such drug hunters should be aware of when developing new drugs. Case studies to illustrate topics like hepatitis C, mRNA vaccines, insulin, and price controls are included to aid in seamless reader comprehension. Written by John LaMattina, former president of Pfizer Global Research and Development and well-known speaker and writer for the pharma industry, sample topics covered and questions explored within the work include: Fiscal consequences of curing hepatitis C mRNA vaccines and the race for a cure Why the government does not deserve a piece of Biopharma’s profits Paying for drugs whose ultimate value is unknown The impact of reduced revenues on R&D This book is a must-read for biopharmaceutical professionals and executives who wish to gain high-level insight into key challenges that must be first understood, then overcome, within the pharmaceutical industry. |
| bluebird sickle cell gene therapy: Translating Gene Therapy to the Clinic Jeffrey Laurence, Michael Franklin, 2014-11-14 Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. - Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application - Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use - Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials |
| bluebird sickle cell gene therapy: Textbook of Personalized Medicine Kewal K. Jain, 2020-12-05 This book is for personalized medicine as a prescription of specific treatments and therapeutics best suited for an individual and considers genetic as well as environmental factors that influence responses to therapy. Best approaches are described for integration of all available technologies for optimizing the therapy of individual patients. This comprehensive third edition covers the latest advances in personalized medicine and several chapters are devoted to various specialties, particulary cancer which is the largest area of application. The book discusses the development of personalized medicine and various players in it such as companies, academic institutions, the government, and the public as the consumer of healthcare. Additionally, the roles of bioinformatics, electronic health records, and digital technologies for personalized medicine are discussed. Textbook of Personalized Medicine, 3rd Edition serves as a convenient source of information for students at many levels and in a wide range of fields, including physicians, scientists, and decision makers in the biopharmaceutical and healthcare industries. |
| bluebird sickle cell gene therapy: Emerging Medical Technologies Gennady Ermak, 2015-08-27 The book provides an overview of new technologies in medicine. It defines each technology and gives an account of its history, and the principles, technical problems, and applications as well as controversies involved. The goal of the book is to provide a comprehensive overview of the emerging medical technologies in a simple way that is understandable to non-specialists. Biomedical science and medical technologies are developing at such a fast pace that it is hard to follow even for specialists. In addition, they have become more and more complex. This book provides readers with a basic understanding of these technologies and helps them to keep up with new developments. The value of the book is that it is comprehensive and yet concise, which makes the learning process fast and easy. |
| bluebird sickle cell gene therapy: Gene Therapy of Cancer Stanton L. Gerson, Edmund C. Lattime, 2002-04-04 The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. - Provides in-depth description of targeted systems and treatment strategies - Explains the underlying cancer biology necessary for understanding a given therapeutic approach - Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses - Presents translational focus with emphasis on requirements for clinical implementation - Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference |
| bluebird sickle cell gene therapy: A Glimpse at Medicine in the Future Mandana Hasanzad, |
| bluebird sickle cell gene therapy: An Introduction to Molecular Biotechnology Michael Wink, 2020-12-03 Completely updated in line with the rapid progress made in the field, this new edition of the highly-praised textbook addresses powerful new methods and concepts in biotechnology, such as genome editing, reprogrammed stem cells, and personalized medicine. An introduction to the fundamentals in molecular and cell biology is followed by a description of standard techniques, including purification and analysis of biomolecules, cloning techniques, gene expression systems, genome editing methods, labeling of proteins and in situ-techniques, standard and high resolution microscopy. The third part focuses on key areas in research and application, ranging from functional genomics, proteomics and bioinformatics to drug targeting, recombinant antibodies and systems biology. The final part looks at the biotechnology industry, explaining intellectual property issues, legal frameworks for pharmaceutical products and the interplay between start-up and larger companies. The contents are beautifully illustrated throughout, with hundreds of full color diagrams and photographs. Provides students and professionals in life sciences, pharmacy and biochemistry with everything they need to know about molecular biotechnology. |
| bluebird sickle cell gene therapy: Sickle Cell Disease in Sub-Saharan Africa Baba Inusa, Kanayo Nwankwo, Nkechikwu Azinge-Egbiri, Bukola Bolarinwa, 2024-04-30 This important collection provides an epidemiological perspective on the continuing scope of sickle cell disease (SCD) in sub-Saharan Africa, alongside the clinical attempts to provide comprehensive care in a resource-limited setting. The book moves from a clinical profile of SCD to screening for the disease and ongoing patient care. There are chapters on pain management, organ failure, infections and transfusions, as well as nutrition and neurocognitive complications. The book concludes with chapters on anti-sickness medication, cell transplantation and nursing care. The first in a two-volume set offering a multi-disciplinary perspective on SCD, this is a comprehensive resource that applies clinical knowledge to the practical challenges faced in sub-Saharan Africa. It will be important reading for medical students taking courses in haematology as well as those studying Public Health in sub-Saharan Africa. Practitioners in the region will also find it invaluable in developing their understanding of this pervasive disease. |
| bluebird sickle cell gene therapy: Heritable Human Genome Editing The Royal Society, National Academy of Sciences, National Academy of Medicine, International Commission on the Clinical Use of Human Germline Genome Editing, 2021-01-16 Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight. |
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TM the longest-studied approved gene therapy - bluebird bio
LYFGENIA is a one-time gene therapy to treat sickle cell disease in patients 12 years of age or older and a history of vaso-occlusive events. Sickle cell disease is a genetic, inherited, lifelong …
HIGHLIGHTS OF PRESCRIBING INFORMATION - bluebird bio
LYFGENIA is an autologous hematopoietic stem cell -based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso …
SICKLE CELL TREATMENT OPTIONS - bluebird bio
ones better navigate sickle cell care. This guide aims to explain the different types of sickle cell treatment options, so that you and your healthcare team can make informed decisions about …
bluebird bio Announces Global Strategic Collaboration with …
bluebird bio is developing potentially transformative gene therapies for severe genetic and orphan diseases. bluebird bio has two clinical-stage programs in development for childhood cerebral …
MEDICATION GUIDE LYFGENIA (pronounced lif-JEN-ee-uh
LYFGENIA is a one-time gene therapy to treat sickle cell disease. Sickle cell disease is a genetic, inherited, lifelong disease caused by an alteration in one of the genes in the red blood cell, the …
HIGHLIGHTS OF PRESCRIBING INFORMATION These …
ZYNTEGLO is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell …
SICKLE CELL UNDERSTANDING - bluebird bio
• MEDICATIONS TO MANAGE PAIN can help people with sickle cell manage acute and chronic pain • BLOOD TRANSFUSIONS can help relieve symptoms of sickle cell and potentially …
bluebird bio Awarded up to $4.2 Million from the French …
Sickle cell anemia is characterized by clotting of improperly shaped red blood cells, which leads to a wide variety of serious health problems including chronic pain and high risk for stroke. Sickle …
bluebird bio Gene Therapy Sample Letter of Intent
• Participation in cell and gene therapy clinical trials • Experience with transplant and currently approved cell and gene therapies • Implementation of a designated autologous cell / gene …
Following treatment for up to 6 months - bluebird bio
ZYNTEGLO is a one-time gene therapy to treat beta-thalassemia (also known as beta-thalassemia major or Cooley’s Anemia) in patients who require regular transfusions. Beta …
