Current Clinical Applications of In Vivo Gene Therapy with AAVs
| Autor: | Jacques P. Tremblay, Jerry R. Mendell, Barry J. Byrne, Shannon E. Boye, Louise R. Rodino-Klapac, Christine N. Kay, Kimberly Goodspeed, Samiah Al-Zaidy, Sanford L. Boye, Steven J. Gray, Lindsey A. George, Stephanie Salabarria, Manuela Corti |
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| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
Strimvelis
Duchenne muscular dystrophy Genetic enhancement Genetic Vectors Gene Expression Review Bioinformatics Choroideremia 03 medical and health sciences 0302 clinical medicine Drug Discovery Retinitis pigmentosa Genetics medicine Animals Humans Molecular Biology 030304 developmental biology Pharmacology 0303 health sciences business.industry Clinical Studies as Topic Genetic Diseases Inborn Spinal muscular atrophy Genetic Therapy Dependovirus medicine.disease Combined Modality Therapy Canavan disease 3. Good health Treatment Outcome Organ Specificity 030220 oncology & carcinogenesis Hereditary Diseases Molecular Medicine business |
| Zdroj: | Molecular Therapy |
| ISSN: | 1525-0024 1525-0016 |
| Popis: | Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable and possibly curative clinical benefit with a single treatment. This review is limited to gene therapy using adeno-associated virus (AAV) because the gene delivered by this vector does not integrate into the patient genome and has a low immunogenicity. There are now five treatments approved for commercialization and currently available, i.e., Luxturna, Zolgensma, the two chimeric antigen receptor T cell (CAR-T) therapies (Yescarta and Kymriah), and Strimvelis (the gammaretrovirus approved for adenosine deaminase-severe combined immunodeficiency [ADA-SCID] in Europe). Dozens of other treatments are under clinical trials. The review article presents a broad overview of the field of therapy by in vivo gene transfer. We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer’s disease, Parkinson’s disease, Canavan disease, aromatic l-amino acid decarboxylase [AADC] deficiency, and giant axonal neuropathy), ocular disorders (Leber congenital amaurosis, age-related macular degeneration [AMD], choroideremia, achromatopsia, retinitis pigmentosa, and X-linked retinoschisis), the bleeding disorder hemophilia, and lysosomal storage disorders. Graphical Abstract Researchers have been trying to develop therapies for hereditary diseases for more than 30 years. Several problems have been successfully resolved and thus Tremblay and colleagues are describing the recent progress of clinical applications of gene therapy. There are now several in vivo treatments under trials, and some approved for commercialization. |
| Databáze: | OpenAIRE |
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