AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
Abstract
Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is characterized by the deterioration of alpha motor neurons in the brainstem and spinal cord. Currently, there is no cure for SMA, which calls for an urgent need to explore affordable and effective therapies and to maximize patients' independence and quality of life. Adeno-associated virus (AAV) vector, one of the most promising and well-investigated vehicles for...
Paper Details
Title
AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
Published Date
Jun 26, 2019
Journal
Volume
26
Issue
7-8
Pages
287 - 295
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