Gene therapy for CNS diseases – Krabbe disease
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چکیده
منابع مشابه
Gene therapy for CNS diseases – Krabbe disease
This is a brief report of the 19th Annual Meeting of the American Society of Gene and Cell Therapy that took place from May 4th through May 7th, 2016 in Washington, DC, USA. While the meeting provided many symposiums, lectures, and scientific sessions this report mainly focuses on one of the sessions on the "Gene Therapy for central nervous system (CNS) Diseases" and specifically on the "Gene T...
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Substantial progress has been made over the last 20 years in understanding the basic biology and function of the normal nervous system, and in elucidating molecular and cellular mechanisms that underlie neurological disease. Together with these advances in understanding have come discoveries of novel genes and proteins, which collectively present an unprecedented opportunity to intervene in and...
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X-linked adrenoleukodystrophy (X-ALD) is a CNS demyelinating disorder caused by loss-of-function mutations in the ABCD1 gene, which encodes the peroxisomal adenosine triphosphate-binding cassette transporter ALD. The ALD transporter imports very-long-chain fatty-acids into peroxisomes where they are degraded. X-ALD manifests often in boys between 5-12 years by progressive cerebral demyelination...
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ژورنال
عنوان ژورنال: BioImpacts
سال: 2016
ISSN: 2228-5660,2228-5652
DOI: 10.15171/bi.2016.09