Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy
نویسندگان
چکیده
منابع مشابه
Perinatal systemic gene delivery using adeno-associated viral vectors
Neurodegenerative monogenic diseases often affect tissues and organs beyond the nervous system. An effective treatment would require a systemic approach. The intravenous administration of novel therapies is ideal but is hampered by the inability of such drugs to cross the blood-brain barrier (BBB) and precludes efficacy in the central nervous system. A number of these early lethal intractable d...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2019
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2018.079