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Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

15 Feb 2018

Abstract excerpt

transgene (AAV9/JeT-GAN) currently employed in the clinical trial. Treatment with AAV/JeT-GAN restored the normal configuration of IFs in patient fibroblasts within days in cell culture and by 4 weeks in GAN KO mice. IT delivery of AAV9/JeT-GAN in aged GAN KO mice preserved sciatic nerve ultrastructure, reduced neuronal IF accumulations and attenuated rotarod dysfunction. This strategy conferred sustained...

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PMID: 29766026
DOI: 10.1016/j.omtm.2018.02.005

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