Dr. Kathy Morelli ’18G publishes culmination of thesis work

Kathy Morelli, 2018 GSBSE graduate and current UCSD postdoctoral fellow, has published the culmination of her thesis work in the Journal of Clinical Investigation.  This paper provides proof-of-concept data for a precedent-setting gene therapy approach for dominant neuromuscular diseases.

Pubmed link below:

Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.

Morelli KH, Griffin LB, Pyne NK, Wallace LM, Fowler AM, Oprescu SN, Takase R, Wei N, Meyer-Schuman R, Mellacheruvu D, Kitzman JO, Kocen SG, Hines TJ, Spaulding EL, Lupski JR, Nesvizhskii A, Mancias P, Butler IJ, Yang XL, Hou YM, Antonellis A, Harper SQ, Burgess RW.

J Clin Invest. 2019 Sep 26. pii: 130600. doi: 10.1172/JCI130600. [Epub ahead of print]

PMID: 31557132

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