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Lentiviral Vectors and Exosomes as Gene and Protein Delivery Tools

Lentiviral Vectors and Exosomes as Gene and Protein Delivery Tools
Author: Maurizio Federico
Publisher: Humana
Total Pages: 276
Release: 2018-05-31
Genre: Medical
ISBN: 9781493981298

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This volume provides current methods and protocols for gene and protein delivery based on both lentivirus-generated and spontaneously released nanovesicles. Lentiviral Vectors and Exosomes as Gene and Protein Delivery Tools guides readers through methods on macromolecule delivery and chapters describe the LV-based protocols of gene engineering. 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 cutting-edge, Lentiviral Vectors and Exosomes as Gene and Protein Delivery Tools aims to ensure successful results in the further study of this vital field.


Lentiviral Vectors

Lentiviral Vectors
Author: Didier Trono
Publisher: Springer Science & Business Media
Total Pages: 276
Release: 2001-12-14
Genre: Medical
ISBN: 9783540421900

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For the first time a compilation of chapters that depict the biological bases underlying the development of lentiviral vectors, the techniques involved in the manufacture of this new gene delivery tool, and its most promising applications.


Lentiviral Vectors and Gene Therapy

Lentiviral Vectors and Gene Therapy
Author: David Escors
Publisher: Springer Science & Business Media
Total Pages: 110
Release: 2012-03-22
Genre: Medical
ISBN: 3034804024

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Gene therapy was conceived during the early and mid part of the 20th century. At first, it was considered a revolutionary biomedical procedure, which could potentially cure any disease for which the molecular bases were understood. Since then, gene therapy has gone through many stages and has evolved from a nearly unrealistic perspective to a real life application. Clinical efficacy in humans was demonstrated at the beginning of this century after its successful application in small-scale clinical trials to cure severe immunodeficiency in children. However, their successes were overshadowed some time later by the occurrence of vector-related leukaemia in a number of treated children. It is in this context that lentiviral vectors have appeared, with improved efficiency and, possibly, increased biosafety. Very recently, the first clinical trials with lentivectors have been carried out with some success. This Brief firstly defines gene therapy, and places lentivectors within this fascinating therapeutic strategy. Then follows a comprehensive description of the development of retroviral and lentiviral vectors and how to specifically target distinct cell types and tissues. The authors also discuss the application of lentivector gene therapy for the treatment of cancer and autoimmune diseases, ending with the application of lentivectors in human gene therapy clinical trials.


A Guide to Human Gene Therapy

A Guide to Human Gene Therapy
Author: Roland W. Herzog
Publisher: World Scientific
Total Pages: 415
Release: 2010
Genre: Medical
ISBN: 9814280917

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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


Molecular Basis and Gene Therapies of Cystic Fibrosis

Molecular Basis and Gene Therapies of Cystic Fibrosis
Author: John Engelhardt
Publisher: MDPI
Total Pages: 208
Release: 2020-12-11
Genre: Medical
ISBN: 303943683X

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Summary of Genes. Thirty years ago, the gene responsible for cystic fibrosis (CF), a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator gene, was identified. This progress has considerably changed our understanding of the pathophysiology of CF and has paved the way for the development of novel and specific therapies for the disease. The CFTR gene contains 27 exons and is characterized by a frequent three base pair deletion of the p.Phe508del. As a result of collaborative work, today more than 2000 mutations have been reported in the gene, and their impact on protein function is now more evident and useful in designing new strategies to correct the gene defect. The field of gene therapy, as illustrated by Ziying Yan in this book, has worked on identifying an efficient vector system for the delivery of the wild-type CFTR gene to the lung. At the same time, animal models have been developed in mice, rats, rabbits, zebrafish, ferrets, and pigs to establish the efficacity of gene delivery. These animals are also of the utmost importance in testing new molecules as modulators or correctors to improve the CFTR lung function. During the last three decades, the epidemiology of CF has dramatically changed, as today cystic fibrosis is now a chronic adult pulmonary disease.


Advances in Parasitology

Advances in Parasitology
Author: David Rollinson
Publisher: Academic Press
Total Pages: 334
Release: 2019-04-26
Genre: Medical
ISBN: 0128177179

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Advances in Parasitology, Volume 104, the latest in a series first published in 1963, contains comprehensive reviews on all areas of interest in contemporary parasitology. The series includes medical studies of parasites of major influence, along with reviews of more traditional areas, such as zoology, taxonomy, and life history, which help to shape current thinking and applications. This new release includes sections on Leishmania Tropica, Extracellular Vesicles in Host-Parasite Interaction, Cathepsins and Vaccines for Fascioliosis, Echinococcosis Transmission on the Tibetan Plateau, A Review of Diagnostics for STH from a Public Health Perspective, and Zoonotic Transmission of Intestinal Parasites: Implications for Control and Elimination. Informs and updates on all the latest developments in the field of parasitology Includes medical studies of parasites of major influence, such as, Leishmania Tropica and the transmission of Echinococcosis Contains contributions from leading authorities and industry experts Features reviews of more traditional areas, such as zoology, taxonomy, and life history, which help to shape current thinking and applications


Recent Advances in Novel Drug Carrier Systems

Recent Advances in Novel Drug Carrier Systems
Author: Ali Demir Sezer
Publisher: BoD – Books on Demand
Total Pages: 516
Release: 2012-10-31
Genre: Medical
ISBN: 9535108107

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This contribution book collects reviews and original articles from eminent experts working in the interdisciplinary arena of novel drug delivery systems and their uses. From their direct and recent experience, the readers can achieve a wide vision on the new and ongoing potentialities of different drug delivery systems. Since the advent of analytical techniques and capabilities to measure particle sizes in nanometer ranges, there has been tremendous interest in the use of nanoparticles for more efficient methods of drug delivery. On the other hand, this reference discusses advances in the design, optimization, and adaptation of gene delivery systems for the treatment of cancer, cardiovascular, pulmonary, genetic, and infectious diseases, and considers assessment and review procedures involved in the development of gene-based pharmaceuticals.


Novel Technologies for Vaccine Development

Novel Technologies for Vaccine Development
Author: Igor S Lukashevich
Publisher: Springer
Total Pages: 393
Release: 2014-11-13
Genre: Medical
ISBN: 3709118182

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This book presents a detailed overview of the development of new viral vector-based vaccines before discussing two major applications: preventive vaccines for infectious diseases and therapeutic cancer vaccines. Viral vector-based vaccines hold a great potential for development into successful pharmaceutical products and several examples at the advanced pre-clinical or clinical stage are presented. Nevertheless, the most efforts were focused on novel and very innovative technologies for new generation of vector-based vaccines. Furthermore, specific topics such as delivery and adjuvant and protection strategies for cell-mediated-based vaccines are presented. Given its scope, the book is a “must read” for all those involved in vaccine development, both in academia and industrial vaccine development.


Gene Therapy in Cancer

Gene Therapy in Cancer
Author: Malcolm K. Brenner
Publisher: CRC Press
Total Pages: 256
Release: 1996-08-21
Genre: Medical
ISBN: 9780824794811

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Integrates critical reviews of the current strategies of gene transfer in cancer, discusses the technologies of gene therapy, and provides the necessary background to interpret the data-offering an objective survey of the advantages and limitations of this therapeutic field to newcomers and established investigators alike.


Viral Vectors for Gene Therapy

Viral Vectors for Gene Therapy
Author: Otto-Wilhelm Merten
Publisher: Humana
Total Pages: 0
Release: 2011-05-19
Genre: Medical
ISBN: 9781617790942

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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.