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 … Advances in Exon Editing for Hemophilia, Spinal Muscular Atrophy, and Cancer TherapyRead more
exon
ExonEdit: Precision Editing of Exons for Therapeutic and Industrial Applications
ExonEdit: Precision Editing of Exons for Therapeutic and Industrial Applications Core Definition Exon: DNA segments within genes that encode proteins, constituting approximately 1.5% of the human genome. ExonEdit (Exon Editing): A biotechnology that precisely modifies exon sequences to regulate prot … ExonEdit: Precision Editing of Exons for Therapeutic and Industrial ApplicationsRead more