Pharmaceutical Biotechnology

Pharmaceutical Biotechnology
Author: Daan J. A. Crommelin
Publisher: CRC Press
Total Pages: 456
Release: 2002-11-14
Genre: Medical
ISBN: 9780415285018

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.

Polymers and Nanomaterials for Gene Therapy

Polymers and Nanomaterials for Gene Therapy
Author: Ravin Narain
Publisher: Woodhead Publishing
Total Pages: 303
Release: 2016-01-09
Genre: Science
ISBN: 0081005210

Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. - Explores current challenges in the research of genetic diseases - Discusses polymers for gene therapy and their function in designing advanced materials - Provides examples of organic and inorganic nanomaterials for gene therapy - Includes labeling, targeting, and assays - Looks at characterization, physico-(bio)chemical properties, and applications

Life with Epidermolysis Bullosa (EB)

Life with Epidermolysis Bullosa (EB)
Author: Jo-David Fine
Publisher: Springer Science & Business Media
Total Pages: 352
Release: 2009-04-02
Genre: Medical
ISBN: 3211792716

Epidermolysis bullosa (EB) is a heterogenous group of genodermatoses characterized by the formation of blisters and erosions on skin and mucous membrans from birth on. The cause are mutations in the genes of structural proteins of the junction between epidermis and dermis. This book deals with the treatment of this skin disease itself and its many extracutaneous complications. There is no previous book which has been focused on the therapy and it will be based on evidence-based data derived from the world ́s largest cohort of inherited EB-patients, the American EB Registry. An important chapter will discuss gene therapy in hereditary EB which has been recently successfully performed within a localized skin site on a single EB patient as a proof-of-principle test. Given its unique collective contents, the monograph will provide the primary source for clinical informations of this oftentimes severe multiorgan disease.

Molecular Biology of the Skin

Molecular Biology of the Skin
Author: Michael Darmon
Publisher: Academic Press
Total Pages: 306
Release: 2012-12-02
Genre: Science
ISBN: 0323139302

Molecular Biology of the Skin: The Keratinocyte comprehensively reviews the major aspects of keratinocyte and epidermal differentiation, physiology, and pathology, primarily focusing on the molecular aspects. This exciting new resource discusses keratin genes, retinoic acid, and the use of transgenic animals in the study of dermatological pathology. The volume also highlights areas of genetic disease, new animal models to help in understanding dermatological disorders, and gene therapy using skin as a target. W.W. Franke, a pioneer in the study of the molecular biology of keratins, has written the foreword for the book.Molecular Biology of the Skin: The Keratinocyte is intended for use by dermatologists and basic researchers in cell and developmental biology. It will also be valuable for surgeons and other clinicians as well as researchers in gene therapy, virology, and pharmacology.* * Reviews keratinocyte (and epidermal) differentiation. physiology, and pathology, focusing on the molecular aspects* -Discusses keratin genes, retinoic acid, and the use of transgenic animals in the study of dermatalogical pathology* -Highlights genetic disease, new animal models, and gene therapy

The Skin and Gene Therapy

The Skin and Gene Therapy
Author: Ulrich R. Hengge
Publisher: Springer Science & Business Media
Total Pages: 336
Release: 2012-12-06
Genre: Medical
ISBN: 3642564410

This first comprehensive guide to the novel field of gene therapy in the skin provides insight into the basic principles and methods of gene transfer into the skin and its applications. It enables the understanding of possibilities and implications such as immunization and treatment as well as safety aspects. Gene therapy in dermatology is the avantgarde of a new field in science with a great future for human health.

Therapeutic Dressings and Wound Healing Applications

Therapeutic Dressings and Wound Healing Applications
Author: Joshua Boateng
Publisher: John Wiley & Sons
Total Pages: 444
Release: 2020-03-09
Genre: Science
ISBN: 1119433266

The latest research on techniques for effective healing of chronic and difficult to heal wounds The healing of chronic wounds is a global medical concern, specifically for patients suffering from obesity and type II diabetes. Therapeutic Dressing and Wound Healing Applications is an essential text for research labs, industry professionals, and general clinical practitioners that want to make the shift towards advanced therapeutic dressing and groundbreaking wound application for better healing. This book takes a clinical and scientific approach to wound healing, and includes recent case studies to highlight key points and areas of improvement. It is divided into two key sections that include insight into the biochemical basis of wounds, as well as techniques and recent advancements. Chapters include information on: ● Debridement and disinfection properties of wound dressing ● Biofilms, silver nanoparticles, and honey dressings ● Clinical perspectives for treating diabetic wounds ● Treating mixed infections ● Wound healing and tissue regeneration treatments ● Gene based therapy, 3D bioprinting and freeze-dried wafers Anyone looking to update and improve the treatment of chronic wounds for patients will find the latest pertinent information in Therapeutic Dressing and Wound Healing Applications.

Somatic Gene Therapy

Somatic Gene Therapy
Author: P.L. Chang
Publisher: CRC Press
Total Pages: 242
Release: 2018-01-17
Genre: Science
ISBN: 1351093665

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.

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

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

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Exploring Novel Clinical Trial Designs for Gene-Based Therapies
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
Total Pages: 127
Release: 2020-08-27
Genre: Medical
ISBN: 0309672988

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.

The Gene Therapy Plan

The Gene Therapy Plan
Author: Mitchell L. Gaynor, MD
Publisher: Penguin Books
Total Pages: 370
Release: 2016-04-05
Genre: Health & Fitness
ISBN: 0143108190

Dr. Mitchell Gaynor, integrative medicine pioneer, details what we can each do individually to keep our bodies healthy.