生产企业：Precision BioSciences, Inc.
PBGENE-DMD employs two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene, aiming to restore a near full-length functional dystrophin protein that more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches.
Duchenne muscular dystrophy (DMD)
共 1 项相关疾病
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