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…
Exon Editing: Advances and Prospects in Genetic Disease and Cancer Therapy Exon editing, an emerging technology that precisely modulates RNA splicing or directly modifies DNA exon sequences to correct pathogenic variants or regulate gene expression, combines innovations in gene editing (e.g., CRISPR…
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…