SIRT2‐knockdown rescues GARS‐induced Charcot‐Marie‐Tooth neuropathy
نویسندگان
چکیده
منابع مشابه
Intrathecal gene therapy rescues a model of demyelinating peripheral neuropathy.
Inherited demyelinating peripheral neuropathies are progressive incurable diseases without effective treatment. To develop a gene therapy approach targeting myelinating Schwann cells that can be translatable, we delivered a lentiviral vector using a single lumbar intrathecal injection and a myelin-specific promoter. The human gene of interest, GJB1, which is mutated in X-linked Charcot-Marie-To...
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ژورنال
عنوان ژورنال: Aging Cell
سال: 2021
ISSN: 1474-9718,1474-9726
DOI: 10.1111/acel.13391