Muscle gene therapy / Dongsheng Duan, Jerry R. Mendell, editors.
| Other author | Duan, Dongsheng, editor. |
| Other author | Mendell, Jerry R. (Jerry Roy), 1942- editor. |
| Format | Electronic |
| Edition | Second edition. |
| Publication | Cham, Switzerland : Springer, [2019] |
| Copyright Date | ©2019 |
| Description | 1 online resource (xvi, 811 pages) : illustrations (some color) |
| Supplemental Content | Direct link to ebook |
| Subjects |
| Contents | Part 1. Foundations for muscle gene therapy. An overview of muscle biology and physiology for muscle gene therapy -- Molecular basis of muscle disease -- Animal models for muscle disease and muscle gene therapy -- Muscle stem cell biology and implication in gene therapy -- Pluripotent stem cells for gene therapy of hereditary muscle disorders -- MicroRNAs (miRs) in muscle gene therapy -- Immune system regulation of muscle injury and disease -- Design of muscle gene therapy expression cassette -- Chapter 9 Non-viral vector mediated muscle gene therapy -- Viral vectors for muscle gene therapy -- Development of next generation muscle gene therapy AAV vectors -- Histological and biochemical evaluation of muscle gene therapy -- Optical polarization tractography imaging of structural changes in the skeletal and cardiac muscles of the mdx4cv mice -- Biomarkers for muscle disease gene therapy -- Large-scale clinical manufacturing of AAV Vectors for systemic muscle gene therapy -- Genome-editing for muscle gene therapy -- Part 2. Preclinical muscle gene therapy. Considerations on preclinical muscle gene therapy studies -- Gene replacement therapy for Duchenne muscular dystrophy -- Recent advances in AON-mediated exon-skipping therapy for Duchenne muscular dystrophy -- AAV-mediated exon-skipping therapy for Duchenne muscular dystrophy -- Alternate translational initiation of dystrophin: a novel therapeutic approach -- Genome editing for Duchenne muscular dystrophy -- Sarcolipin knockdown therapy for Duchenne muscular dystrophy -- Gene therapy for central nervous system in Duchenne muscular dystrophy -- Therapeutic approach for dysferlinopathy in animal models -- Muscle cell membrane repair and therapeutic implications -- Treatment of muscular dystrophy-dystroglycanopathy: unlocking the potential of genetic engineering -- RNAi therapy for dominant muscular dystrophies and other myopathies -- Gene therapy for facioscapulohumeral muscular dystrophy (FSHD) -- Gene therapy and gene editing for myotonic dystrophy -- Gene therapy for oculopharyngeal muscular dystrophy -- Gene therapy for X-linked myotubular myopathy -- Pre-clinical gene therapy studies for metabolic myopathy -- Elimination of mutant mitochondrial DNA in mitochondrial myopathies using gene editing enzymes -- Gene therapy for Charcot-Marie-Tooth inherited neuropathy -- Muscle -- a potent target in vaccination -- Part 3. Clinical muscle gene therapy. Patient and family perspective on muscle gene therapy -- Design of clinical trials for gene therapy in muscular dystrophy -- Path to clinical trials: trial design, development of the clinical product and safety concerns in the implementation of clinical trials -- Muscle MRI as an endpoint in clinical trials -- Gene therapy clinical trials for Duchenne and limb girdle muscular dystrophies lessons learned -- Duchenne muscular dystrophy exon-skipping trials -- What we have learned from 10 years of DMD exon skipping trials -- Clinical gene therapy trials for Pompe disease -- Muscle-directed gene therapy for alpha-1 antitrypsin deficiency. |
| Abstract | "Muscle disease represents an important health threat to the general population. Unlike diseases affecting other tissues/organs, therapeutic options are quite limited for many inherited muscle diseases such as Duchenne muscular dystrophy. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. A variety of new strategies, such as exon-skipping and RNAi, have greatly expanded the scope of muscle gene therapy beyond the traditional gene replacement approach. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy"--Publisher's description |
| Bibliography note | Includes bibliographical references and index. |
| Source of description | Online resource; title from electronic title page (ProQuest Ebook Central, viewed on February 4, 2021). |
| Issued in other form | Print version: Muscle gene therapy. Second edition. Cham : Springer, 2019 9783030030940 |
| ISBN | 9783030030957 (electronic book) |
| ISBN | 3030030954 (electronic book) |
| Standard identifier# | 10.1007/978-3-030-03095-7 |
| Stock number | com.springer.onix.9783030030957 Springer Nature |
Availability
| Library | Location | Call Number | Status | Item Actions |
|---|---|---|---|---|
| Electronic Resources | Access Content Online | ✔ Available |