AAV-vectored base editor trans-splicing delivers dystrophin repair

Li, Z; Gonçalves, MAFV

Gonçalves, MAFV (通讯作者),Leiden Univ, Dept Cell & Chem Biol, Med Ctr, Einthovenweg 20, NL-2333 ZC Leiden, Netherlands.

MOLECULAR THERAPY-NUCLEIC ACIDS, 2023; 32 (): 900