Advances in Exon Editing for Hemophilia, Spinal Muscular Atrophy, and Cancer Therapy
Advances in Exon Editing for Hemophilia, Spinal Muscular Atrophy, and Cancer Therapy (As of May 2025) I. Spinal Muscular Atrophy (SMA): Restoring SMN Protein Expression 1. Precision Repair of SMN2 Exon 7 Base Editing: A-to-G editing at the SMN2 exon 7 C6T mutation increases splicing efficiency to 99…
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2025年5月23日
