Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C %26gt; T and c.639+919G %26gt; A GLA Deep Intronic Mutations

作者:Ferri Lorenzo; Covello Giuseppina; Caciotti Anna; Guerrini Renzo; Denti Michela Alessandra; Morrone Amelia
来源:Molecular Therapy - Nucleic Acids, 2016, 5: e380.
DOI:10.1038/mtna.2016.88
  • 出版日期2016-10-25