Gene And Cell Therapies For Beta Globinopathies

Author : Punam Malik
Publisher : Springer
ISBN 13 : 1493972995
Total Pages : 248 pages
Book Rating : 4.99/5 ( download)

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Download or read book Gene and Cell Therapies for Beta-Globinopathies written by Punam Malik and published by Springer. This book was released on 2017-11-09 with total page 248 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Author : Daniel E. Bauer
Publisher : Elsevier Health Sciences
ISBN 13 : 0323546692
Total Pages : pages
Book Rating : 4.90/5 ( download)

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Download or read book Gene Therapy, An Issue of Hematology/Oncology Clinics of North America, E-Book written by Daniel E. Bauer and published by Elsevier Health Sciences. This book was released on 2017-09-27 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Author : Nirali N. Shah
Publisher : Elsevier Health Sciences
ISBN 13 : 0323987761
Total Pages : 273 pages
Book Rating : 4.69/5 ( download)

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Download or read book Gene-Based Therapies for Pediatric Blood Diseases, An Issue of Hematology/Oncology Clinics of North America, E-Book written by Nirali N. Shah and published by Elsevier Health Sciences. This book was released on 2022-07-19 with total page 273 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this issue of Hematology/Oncology Clinics, guest editors Drs. Sung-Yun Pai and Nirali N. Shah bring their considerable expertise to the topic of Gene-Based Therapies for Pediatric Blood Diseases. Top experts in the field cover key topics such as CAR T-cell therapy: current status; engineered T cells; NK-cell therapy; hemoglobinopathies: beta-thalassemia, sickle cell disease; hemophilia A/B; primary immunodeficiencies; and more. Contains 14 relevant, practice-oriented topics including the evolution of gene therapy; viral vectors in hematopoietic stem cell gene therapy; gene editing in hematopoietic stem cells; nonintegrating vectors and engineered capsids; regulatory aspects of gene therapy; and more. Provides in-depth clinical reviews on gene-based therapies for pediatric blood diseases, 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.

Author :
Publisher : ScholarlyEditions
ISBN 13 : 1464904715
Total Pages : 59 pages
Book Rating : 4.14/5 ( download)

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Download or read book Sickle Cell Anemia: New Insights for the Healthcare Professional: 2011 Edition written by and published by ScholarlyEditions. This book was released on 2012-01-09 with total page 59 pages. Available in PDF, EPUB and Kindle. Book excerpt: Sickle Cell Anemia: New Insights for the Healthcare Professional: 2011 Edition is a ScholarlyBrief™ that delivers timely, authoritative, comprehensive, and specialized information about Sickle Cell Anemia in a concise format. The editors have built Sickle Cell Anemia: New Insights for the Healthcare Professional: 2011 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Sickle Cell Anemia in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Sickle Cell Anemia: New Insights for the Healthcare Professional: 2011 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.

Author :
Publisher : Elsevier Health Sciences
ISBN 13 : 0323722628
Total Pages : pages
Book Rating : 4.29/5 ( download)

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Download or read book Advances in Pediatrics, E-Book 2020 written by and published by Elsevier Health Sciences. This book was released on 2020-08-07 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: Each year, Advances in Pediatrics focuses on providing current clinical information on important topics in pediatrics. Dr. Carol Berkowitz and her editorial board, comprised of top experts in the field, have assembled authors to provide updates on the following topics: Evaluation and Management of Febrile Infants; Pediatric Emergency Medicine and Ultrasonography; The Patient-Centered Pediatric Emergency Department; Health Considerations of Refuge and Immigrant Children; Management of scoliosis; Health and Wellness for LGBTQ Youth; Sexually exploited children: recognizing and addressing; Movement disorders in children; Childhood trauma management in primary care; Feeding issues in young children; Physician Well-being and Burnout; New Molecular Methods for Diagnosing Infectious Diseases; Parental refusal: treatments, procedures and vaccines; Pediatric oncology in the ICU setting; Diaphragmatic hernia: Management and Outcomes; and Global Health and Pediatric Education: Opportunities and Challenges. Readers will come away with the clinical information that supplements their professional knowledge so they can make informed clinical decisions that improve patient outcomes.

Author : Denise Harmening
Publisher : F.A. Davis
ISBN 13 : 1719653135
Total Pages : 2579 pages
Book Rating : 4.38/5 ( download)

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Download or read book Clinical Hematology and Fundamentals of Hemostasis written by Denise Harmening and published by F.A. Davis. This book was released on 2024-02-01 with total page 2579 pages. Available in PDF, EPUB and Kindle. Book excerpt: A full-color text, lab manual, & atlas—all in one! Here are all the tools medical laboratory science students need to master the principles of hematology and the fundamentals of hemostasis. Author Denise M. Harmening has curated contributions from a team of expert educators and clinicians. With support from her Associate Editor LeAnne Hutson, she brings you comprehensive, yet focused coverage that prepares you for the real world in which you will practice. Begin with an introduction to clinical hematology and the anemias, and then progress through white blood cell disorders and hemostasis to thrombosis and laboratory methods. Find step-by-step laboratory procedures and critical-thinking cases online at FADavis.com for easy access anytime, anywhere.

Author :
Publisher : ScholarlyEditions
ISBN 13 : 1464909628
Total Pages : 28 pages
Book Rating : 4.27/5 ( download)

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Download or read book Lentivirus: New Insights for the Healthcare Professional: 2011 Edition written by and published by ScholarlyEditions. This book was released on 2012-01-09 with total page 28 pages. Available in PDF, EPUB and Kindle. Book excerpt: Lentivirus: New Insights for the Healthcare Professional: 2011 Edition is a ScholarlyPaper™ that delivers timely, authoritative, and intensively focused information about Lentivirus in a compact format. The editors have built Lentivirus: New Insights for the Healthcare Professional: 2011 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Lentivirus in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Lentivirus: New Insights for the Healthcare Professional: 2011 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.

Author : Elliot Vichinsky
Publisher : Elsevier Health Sciences
ISBN 13 : 0323290000
Total Pages : 281 pages
Book Rating : 4.05/5 ( download)

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Download or read book Emerging Therapies Targeting the Pathophysiology of Sickle Cell Disease, An Issue of Hematology/Oncology Clinics, written by Elliot Vichinsky and published by Elsevier Health Sciences. This book was released on 2014-05-23 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: This issue of Hematology/Oncology Clinics, guest edited by Dr. Elliott Vichinsky, is devoted to Sickle Cell Disease, and focuses on pathophysiology of hemoglobinopathies, therapeutic targets, and new approaches to correcting ineffective erythropoiesis and iron dysregulation. Articles in this issue include Polymerization and red cell membrane changes; Overview on reperfusion injury in the pathophysiology of SCD; Regulation of ineffective erythropoiesis in iron metabolism; Altering oxygen affinity; Cellular adhesion and the endothelium; Arginine therapy; Role of the hemostatic system on SCD pathophysiology and potential therapeutics; Adenosine signaling and novel therapies; New approaches to correcting ineffective erythropoiesis and iron dysregulation; New approaches to correcting ineffective erythropoiesis and iron dysregulation; Fetal hemoglobin induction; Gene therapy for hemoglobinopathies; and Oxidative injury and the role of antioxidant therapy.

Author : Maria Cristina Galli
Publisher : Springer Nature
ISBN 13 : 3031345673
Total Pages : 243 pages
Book Rating : 4.78/5 ( download)

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Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer Nature. This book was released on 2023-08-01 with total page 243 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization. This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs. Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, as well as pertinent ‘must-know’ guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: a Global Perspective is part of the American Society of Gene & Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Author : Cynthia D. Brown
Publisher : Springer Nature
ISBN 13 : 3030686884
Total Pages : 169 pages
Book Rating : 4.88/5 ( download)

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Download or read book Transitioning Care from Pediatric to Adult Pulmonology written by Cynthia D. Brown and published by Springer Nature. This book was released on 2021-04-28 with total page 169 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book addresses an unmet need in the care of adolescents and young adults with lung disease. The increasing survival of young adults with childhood-onset pulmonary conditions is a testament to major advances in treatments and health care delivery. With the increase in survival of children with chronic respiratory conditions, there is a need for formalized transition programs to integrate adolescents and young adults into the adult model of care. This book helps fill gaps in knowledge to best achieve that initiative. This book takes a comprehensive approach to transition care in pulmonary medicine by satisfying the following objectives: 1) Understand the barriers and developmental challenges in the transition from pediatric to adult care for patients with chronic childhood-onset pulmonary disease; 2) Learn about successful evidence based transition models in pulmonary disease populations, focusing on key process and outcome measures for success; and 3) Develop knowledge to design, implement, and measure a transition program based on evidence and expert opinion. In the first section, the book outlines general principles of transition care that are applicable to all patients regardless of underlying disease process and describe best practices for performing necessary research in transition care. In the second section, the book explores psychosocial factors known to play a role in affecting transition outcomes, including parental support, psychological development, and socioeconomic factors. In the final section, transition outcomes and best practices in specific respiratory diseases are outlined. With a focus on populations with chronic pulmonary disease, this book highlights the challenges and barriers of transition, reviews model systems to understand the essential components of a transition program, and provides evidence-based information to navigate these barriers and achieve successful outcomes during transition to adulthood. This is an ideal guide for pediatric and adult pulmonary providers caring for patients who are transition age, as well as multidisciplinary care team members who work with these providers in care models on transition projects to improve the transition process.