Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

An NIH-sponsored phase I clinical trial is underway to test a potential treatment for giant axonal neuropathy (GAN) using viral-mediated GAN gene replacement (https://clinicaltrials.gov/ct2/show/NCT02362438). This trial marks the first instance of intrathecal (IT) adeno-associated viral (AAV) gene t...

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Bibliographic Details
Main Authors: Rachel M. Bailey, Diane Armao, Sahana Nagabhushan Kalburgi, Steven J. Gray
Format: Article
Language:English
Published: Elsevier 2018-06-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050118300172

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http://www.sciencedirect.com/science/article/pii/S2329050118300172

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