Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH
单次静脉注射AAVHSC15-PAH后,苯丙酮尿症小鼠模型得到持续纠正
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2020.03.009
Ahmed, Seemin S; Rubin, Hillard; Wang, Minglun; Faulkner, Deiby; Sengooba, Arnold; Dollive, Serena N; Avila, Nancy; Ellsworth, Jeff L; Lamppu, Diana; Lobikin, Maria; Lotterhand, Jason; Adamson-Small, Laura; Wright, Teresa; Seymour, Albert; Francone, Omar L