Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal
婴儿恶性骨硬化症的基因治疗:临床前研究综述及表型逆转的概念验证
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2020.12.009
Moscatelli, Ilana; Almarza, Elena; Schambach, Axel; Ricks, David; Schulz, Ansgar; Herzog, Christopher D; Henriksen, Kim; Askmyr, Maria; Schwartz, Jonathan D; Richter, Johan