Gene Therapy for Renal Diseases and Transplantation


Book Description

Initially conceived as a strategy to remedy inherited genetic disorders, gene therapy has been successfully applied in the last decade to ameliorate the renal function compromised by progressive renal diseases and to prevent kidney allograft rejection in experimental animals. In the present book, world-renowned experts are presenting new insights into viral and non-viral systems used to effect gene delivery, one chapter being dedicated to the new field of RNA interference (RNAi). This latter method may be successfully used in renal ischemia-reperfusion injury, trauma and transplantation. In the near future, gene therapy might also prove to be a new strategy to target molecules involved in tissue damage and inflammation processes that underlie ARF. So far, gene transfer has been successfully applied in experimental glomerulonephritis and interstitial fibrosis, and studies in larger animals are keenly awaited. Also covered are issues related to transplantation, which is the therapy of choice in many end-stage organ failures. Transfer of genes whose protein products have immunomodulatory properties have proven beneficial in treating acute and chronic graft rejection, one of the problems not satisfactorily solved by current anti-rejection drugs. Gene therapy thus may become a reality in clinical transplantation once its efficacy in larger animals has been demonstrated. Last but not least, a possible benefit of targeted gene therapy in renal cancer or HIV-associated nephropathy is explored. Covering a wide spectrum of topics, this publication provides a valuable overview of current developments and issues.




Continuous Renal Replacement Therapy


Book Description

Continuous Renal Replacement Therapy provides concise, evidence-based, bedside guidance for the management of critically ill patients with acute renal failure, offering quick reference answers to clinicians' questions about treatments and situations encountered in daily practice.




Exploring Novel Clinical Trial Designs for Gene-Based Therapies


Book Description

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.




Disease Control Priorities, Third Edition (Volume 5)


Book Description

Cardiovascular, respiratory, and related conditions cause more than 40 percent of all deaths globally, and their substantial burden is rising, particularly in low- and middle-income countries (LMICs). Their burden extends well beyond health effects to include significant economic and societal consequences. Most of these conditions are related, share risk factors, and have common control measures at the clinical, population, and policy levels. Lives can be extended and improved when these diseases are prevented, detected, and managed. This volume summarizes current knowledge and presents evidence-based interventions that are effective, cost-effective, and scalable in LMICs.




Kidney Transplantation


Book Description







Immunopharmacology


Book Description

During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.




A Guide to Human Gene Therapy


Book Description

1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne




Pediatric Renal Transplantation


Book Description

A detailed, contributed reference offering broad coverage of renal transplantation in children. Diagnosis, the patient's medical management, operative methods, surgical and medical complications, donor selection, immunosuppression, late effects on growth and development and psychosocial factors are among the topics discussed. Features a chapter on how to set up and manage a pediatric renal transplant program.




Regulatory Aspects of Gene Therapy and Cell Therapy Products


Book Description

This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). 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 for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, 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 and 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.