Article
Transplanted human neural stem cells rescue phenotypes in zQ175 Huntington's disease mice and innervate the striatum.
Molecular therapy : the journal of the American Society of Gene Therapy - 6 Dec 2023
Holley Sandra M, Reidling Jack C, Cepeda Carlos, Wu Jie, Lim Ryan G, Lau Alice, Moore Cindy, Miramontes Ricardo, Fury Brian, Orellana Iliana, Neel Michael, Coleal-Bergum Dane, Monuki Edwin S, Bauer Gerhard, Meshul Charles K, Levine Michael S, Thompson Leslie M
Abstract excerpt
Huntington's disease (HD), a genetic neurodegenerative disorder, primarily affects the striatum and cortex with progressive loss of medium-sized spiny neurons (MSNs) and pyramidal neurons, disrupting cortico-striatal circuitry. A promising regenerative therapeutic strategy of transplanting human neural stem cells (hNSCs) is challenged by the need for long-term functional integration. We previously described that,...
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