Life-limiting peripheral organ dysfunction in feline Sandhoff disease emerges after effective CNS gene therapy.

Aime K Johnson,Victoria J McCurdy,Heather L Gray-Edwards,Anne S Maguire,J Nicholas Cochran,Amanda L Gross, Haleigh E Skinner,Ashley N Randle,Jamie L Shirley,Brandon L Brunson,Allison M Bradbury,Stanley G Leroy,Misako Hwang,Hannah E Rockwell,Nancy R Cox,Henry J Baker,Thomas N Seyfried,Miguel Sena-Esteves,Douglas R Martin

Annals of neurology（2023）

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摘要

The vector formulation in the current study effectively treats neuropathology in feline Sandhoff disease, but whole-body targeting will be an important consideration in next-generation approaches. This article is protected by copyright. All rights reserved.

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