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Locanabio Presents Preclinical Data from its Vectorized snRNA Exon Skipping Program for DMD and Cas13d Multi-targeting Program for C9orf72 ALS at the American Society of Gene and Cell Therapy (ASGCT)
LBIO-115 demonstrates robust exon 51 skipping resulting in high levels of dystrophin restoration in patient-derived muscle cells and an in vivo DMD disease model Cas13d Multi-targeting efficiently knocks down both sense and antisense toxic RNAs and decreases dipeptide repeats in C9orf72 ALS patient fibroblasts and an in vivo disease model SAN DIEGO, May 16, 2023 […] The post Locanabio Presents Preclinical Data from its Vectorized snRNA Exon Skipping Program for DMD and Cas13d Multi-targeting Pro...
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Posted: May 16 2023, 10:00
Author Name: forextv
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