RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model

Aimiuwu, OV; Fowler, AM; Sah, M; Teoh, JJ; Kanber, A; Pyne, NK; Petri, S; Rosenthal-Weiss, C; Yang, M; Harper, SQ; Frankel, WN

Frankel, WN (corresponding author), Columbia Univ, Irving Med Ctr, Inst Genom Med, 701 West 168th St, New York, NY 10032 USA.; Frankel, WN (corresponding author), Columbia Univ, Irving Med Ctr, Dept Genet & Dev, 701 West 168th St, New York, NY 10032 USA.

MOLECULAR THERAPY, 2020; 28 (7): 1706

Abstract

Developmental and epileptic encephalopathy (DEE) associated with de novo variants in the gene encoding dynamin-1 (DNM1) is a severe debilitating disea......

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