403. Engineering Micro-Dystrophin AAV Vector for Clinical Translation
نویسندگان
چکیده
منابع مشابه
The AAV vector toolkit: poised at the clinical crossroads.
The discovery of naturally occurring adeno-associated virus (AAV) isolates in different animal species and the generation of engineered AAV strains using molecular genetics tools have yielded a versatile AAV vector toolkit. Promising results in preclinical animal models of human disease spurred the much awaited transition toward clinical application, and early successes in phase I/II clinical t...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2015
ISSN: 1525-0016
DOI: 10.1016/s1525-0016(16)34012-6