Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia
نویسندگان
چکیده
منابع مشابه
Systemic gene delivery to the central nervous system using Adeno-associated virus
Adeno-associated virus (AAV)-mediated gene delivery has emerged as an effective and safe tool for both preclinical and clinical studies of neurological disorders. The recent discovery that several serotypes are able to cross the blood-brain barrier when administered systemically has been a real breakthrough in the field of neurodegenerative diseases. Widespread transgene expression after system...
متن کاملLong-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy
Methylmalonic acidemia (MMA) is an organic acidemia caused by deficient activity of the mitochondrial enzyme methylmalonyl-CoA mutase (MUT). This disorder is associated with lethal metabolic instability and carries a poor prognosis for long-term survival. A murine model of MMA that replicates a severe clinical phenotype was used to examine the efficacy of recombinant adeno-associated virus (rAA...
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Gene therapy for cancer offers novel treatment paradigms that will eventually lead to the destruction of tumor cells in patients with solid and hematopoietic malignancies. Major cancer gene therapy approaches that directly target tumor cells include chemosensitization, cytokine gene transfer, inactivation of proto-oncogene expression, replacement of defective tumor suppressor genes, and transdu...
متن کاملGene therapy using adeno-associated virus vectors.
SUMMARY The unique life cycle of adeno-associated virus (AAV) and its ability to infect both nondividing and dividing cells with persistent expression have made it an attractive vector. An additional attractive feature of the wild-type virus is the lack of apparent pathogenicity. Gene transfer studies using AAV have shown significant progress at the level of animal models; clinical trials have ...
متن کاملMethylmalonic acidemia
The authors provide an overview of the hereditary methylmalonic acidemias, a group of metabolic disorders with varied clinical presentations. This includes the most severe form of L-methylmalonyl-CoA mutase deficiency, termed mut(o) methylmalonic acidemia, which, together with the less severe deficiencies of L-methylmalonyl-CoA mutase, are the most common causes of methylmalonic acidemia. They ...
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ژورنال
عنوان ژورنال: Molecular Therapy - Methods & Clinical Development
سال: 2021
ISSN: 2329-0501
DOI: 10.1016/j.omtm.2021.04.005