Gene Therapy Methods

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Viral Vectors for Gene Therapy

Fredric P. Manfredsson,Matthew J. Benskey
Viral Vectors for Gene Therapy Book PDF

Author : Fredric P. Manfredsson,Matthew J. Benskey
Publisher : Humana Press
Page : 328 pages
File Size : 50,8 Mb
Release : 2019-02-01
Category : Medical
ISBN : 1493990640

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Viral Vectors for Gene Therapy by Fredric P. Manfredsson,Matthew J. Benskey Pdf

This volume discusses protocols, ranging from vector production to delivery methods, used to execute gene therapy applications. Chapters are divided into four parts, and cover topics such as design, construction, and application of transcription activation-like effectors; multi-modal production of adeno-associated virus; construction of oncolytic herpes simplex virus; AAV-mediated gene delivery to the mouse liver; and intrathecal delivery of gene therapeutics by direct lumbar puncture in mice. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Comprehensive and authoritative, Viral Vectors for Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students looking to utilize viral vectors in gene therapy experiments.

Gene Therapy Methods

M. Ian Phillips
Gene Therapy Methods Book PDF

Author : M. Ian Phillips
Publisher : Academic Press
Page : 770 pages
File Size : 48,7 Mb
Release : 2002-02-22
Category : Medical
ISBN : 9780121822477

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Gene Therapy Methods by M. Ian Phillips Pdf

This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease

Viral Vectors for Gene Therapy

Otto-Wilhelm Merten,Mohamed Al-Rubeai
Viral Vectors for Gene Therapy Book PDF

Author : Otto-Wilhelm Merten,Mohamed Al-Rubeai
Publisher : Humana
Page : 0 pages
File Size : 49,5 Mb
Release : 2011-05-19
Category : Medical
ISBN : 161779094X

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Viral Vectors for Gene Therapy by Otto-Wilhelm Merten,Mohamed Al-Rubeai Pdf

The huge potential for gene therapy to cure a wide range of diseases has led to high expectations and a great increase in research efforts in this area, particularly in the study of delivery via viral vectors, widely considered to be more efficient than DNA transfection. In Viral Vectors for Gene Therapy: Methods and Protocols, experts in the field present a collection of their knowledge and experience featuring methodologies that involve virus production, transferring protocols, and evaluating the efficacy of gene products. While thoroughly covering the most popular viral vector systems of adenovirus, retrovirus, and adeno-associated virus, this detailed volume also explores less common viral vector systems such as baculovirus, herpes virus, and measles virus, the growing interest in which is creating a considerable demand for large scale manufacturing and purification procedures. Written in the highly successful Methods in Molecular BiologyTM series format, many chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and vital tips on troubleshooting and avoiding known pitfalls. Comprehensive and practical, Viral Vectors for Gene Therapy: Methods and Protocols provides basic principles accessible to scientists from a wide variety of backgrounds for the development of gene therapy viral products that are safe and effective.

Gene Therapy for Cancer

Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Gene Therapy for Cancer Book PDF

Author : Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher
Publisher : Springer Science & Business Media
Page : 469 pages
File Size : 46,6 Mb
Release : 2007-10-26
Category : Medical
ISBN : 9781597452229

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Gene Therapy for Cancer by Kelly K. Hunt,Stephan A. Vorburger,Stephen G. Swisher Pdf

The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.

Gene Therapeutics

Jon A. Wolff
Gene Therapeutics Book PDF

Author : Jon A. Wolff
Publisher : Springer Science & Business Media
Page : 431 pages
File Size : 49,8 Mb
Release : 2012-12-06
Category : Medical
ISBN : 9781468468229

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Gene Therapeutics by Jon A. Wolff Pdf

During the first half century of genetics, coinciding with the first half of this cen tury, geneticists dreamt of the repair of genetic disease by altering or replacing defective genes. H. J. Muller wrote of the great advantages of mutations, "nanoneedles" in his apt term, for delicately probing physiological and chemical processes. In the same spirit, genes could be used to provide treatments of needle point delicacy. Yet, during this period no realistic possibility appeared; it remained but a dream. The situation changed abruptly at the half century. Microbial genetics and its offshoot, cell culture genetics, provided the route. Pneumococcus transformation showed that exogenous DNA could become a permanent part of the genome; yet attempts to reproduce this in animals produced a few tantalizing hints of success, but mostly failures. Transduction, using a virus as mediator, offered a better op portunity. The fITSt reproducible in vivo gene therapy in a whole animal came in 1981. This was in Drosophila, with a transposable element as carrier. Flies were "cured" of a mutant eye color by incorporation of the normal allele, and the effect was transmissible, foreshadowing not only somatic, but germ line gene therapy. At the same time, retroviruses carrying human genes were found to be ex tremely efficient in transferring their contents to the chromosomes of cultured cells.

Gene Therapy Protocols

Paul D. Robbins
Gene Therapy Protocols Book PDF

Author : Paul D. Robbins
Publisher : Humana PressInc
Page : 432 pages
File Size : 55,7 Mb
Release : 1997
Category : Medical
ISBN : 0896034844

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Gene Therapy Protocols by Paul D. Robbins Pdf

Thirty protocols by leading researchers describe in detail all the essential molecular methods for working with gene transfer systems, along with the methods for gene transfer to specific tissue types either in vivo or ex vivo. The easily reproducible methods range from those for specific viral and nonviral for both genetic and acquired diseases, to those concerned with gene delivery to particular tissues. Methods for applying specific therapeutic systems, such as ribozymes and tumor supressor genes for the treatment of AIDS and cancer, are also included in this authoritative collection.

Fast Facts: Gene Therapy

Roland Herzog,Linda Popplewell
Fast Facts Gene Therapy Book PDF

Author : Roland Herzog,Linda Popplewell
Publisher : Karger Medical and Scientific Publishers
Page : 102 pages
File Size : 43,5 Mb
Release : 2020-04-08
Category : Medical
ISBN : 9783318066661

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Fast Facts: Gene Therapy by Roland Herzog,Linda Popplewell Pdf

Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments

Gene Therapy for Neurological Disorders

Fredric P. Manfredsson
Gene Therapy for Neurological Disorders Book PDF

Author : Fredric P. Manfredsson
Publisher : Humana
Page : 0 pages
File Size : 44,5 Mb
Release : 2015-11-26
Category : Medical
ISBN : 1493932705

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Gene Therapy for Neurological Disorders by Fredric P. Manfredsson Pdf

This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course of experimentation and will answer questions such as: What delivery vehicle do you use?; How will you ensure that your vector retains stability?; What expression system best fits your needs?; What route will you choose to deliver your gene therapy agent?; How will you model the neurodegenerative disorder that you aim to investigate and what are the proven methods to treat these disorders in preclinical models? Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols and tips on troubleshooting and avoiding known pitfalls. Authoritative and thorough, Gene Therapy for Neurological Disorders: Methods and Protocols, is a compilation of protocols and instructive chapters intended to give researchers, clinicians, and students of all levels, a foundation upon which future gene therapy experiments can be designed.

Pharmaceutical Biotechnology

Daan J. A. Crommelin,Robert D. Sindelar
Pharmaceutical Biotechnology Book PDF

Author : Daan J. A. Crommelin,Robert D. Sindelar
Publisher : CRC Press
Page : 456 pages
File Size : 50,9 Mb
Release : 2002-11-14
Category : Medical
ISBN : 0415285011

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Pharmaceutical Biotechnology by Daan J. A. Crommelin,Robert D. Sindelar Pdf

The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.

Translating Gene Therapy to the Clinic

Jeffrey Laurence,Michael Franklin
Translating Gene Therapy to the Clinic Book PDF

Author : Jeffrey Laurence,Michael Franklin
Publisher : Academic Press
Page : 347 pages
File Size : 53,8 Mb
Release : 2014-11-14
Category : Science
ISBN : 9780128005644

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Translating Gene Therapy to the Clinic by Jeffrey Laurence,Michael Franklin Pdf

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

Viral Vectors for Gene Therapy

Curtis A. Machida
Viral Vectors for Gene Therapy Book PDF

Author : Curtis A. Machida
Publisher : Springer Science & Business Media
Page : 591 pages
File Size : 53,7 Mb
Release : 2008-02-02
Category : Medical
ISBN : 9781592593040

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Viral Vectors for Gene Therapy by Curtis A. Machida Pdf

Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdoctoral training. The “Notes” section contained in each chapter provides valuable troublesho- ing guides to help develop working protocols for your laboratory. With Viral Vectors for Gene Therapy: Methods and Protocols, it has been my intent to develop a comprehensive collection of modern molecular methods for the construction, development, and use of viral vectors for gene transfer and gene therapy. I would like to thank the many chapter authors for their contributions. They are all experts in various aspects of viral vectors, and I appreciate their efforts and hard work in developing comprehensive chapters. As editor, it has been a privilege to preview the development of Viral Vectors for Gene Therapy: Methods and Protocols, and to acquire insight into the various methodological approaches from the many different contri- tors.

A Guide to Human Gene Therapy

Roland W. Herzog,Sergei Zolotukhin
A Guide to Human Gene Therapy Book PDF

Author : Roland W. Herzog,Sergei Zolotukhin
Publisher : World Scientific
Page : 415 pages
File Size : 43,9 Mb
Release : 2010
Category : Medical
ISBN : 9789814280914

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A Guide to Human Gene Therapy by Roland W. Herzog,Sergei Zolotukhin Pdf

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

Adenoviral Vectors for Gene Therapy

David T. Curiel
Adenoviral Vectors for Gene Therapy Book PDF

Author : David T. Curiel
Publisher : Academic Press
Page : 868 pages
File Size : 52,5 Mb
Release : 2016-03-10
Category : Science
ISBN : 9780128005101

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Adenoviral Vectors for Gene Therapy by David T. Curiel Pdf

Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Cardiac Gene Therapy

Kiyotake Ishikawa
Cardiac Gene Therapy Book PDF

Author : Kiyotake Ishikawa
Publisher : Springer Nature
Page : 342 pages
File Size : 41,7 Mb
Release : 2022-08-30
Category : Science
ISBN : 9781071627075

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Cardiac Gene Therapy by Kiyotake Ishikawa Pdf

This second edition volume expands on the previous edition with updated techniques and discussions on topics such as gene suppression, editing, and reprogramming; cardiac gene therapy vectors and promoters; cardiac gene delivery methods; pulmonary hypertension; and patient screening and measuring the efficacy of cardiac gene therapy. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Cutting-edge and comprehensive, Cardiac Gene Therapy: Methods and Protocols, Second Edition is a valuable tool for researchers in cardiology who are conducting gene therapy research. This book will lead to further advancements and successful clinical translations in the field. Chapter “Direct Reprogramming of Adult Human Cardiac Fibroblasts into Induced Cardiomyocytes Using miRcombo” is available open access under a Creative Commons Attribution 4.0 International License via link.springer.com.

Somatic Gene Therapy

P.L. Chang
Somatic Gene Therapy Book PDF

Author : P.L. Chang
Publisher : CRC Press
Page : 313 pages
File Size : 51,9 Mb
Release : 2018-01-17
Category : Science
ISBN : 9781351085212

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Somatic Gene Therapy by P.L. Chang Pdf

As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.