作者
NULL AUTHOR_ID,NULL AUTHOR_ID,NULL AUTHOR_ID,Eeuwe Dijk,NULL AUTHOR_ID,Shaoliang Yu,NULL AUTHOR_ID,NULL AUTHOR_ID,Elizabeth J. English,NULL AUTHOR_ID,NULL AUTHOR_ID,Matthew S Benczkowski,NULL AUTHOR_ID,NULL AUTHOR_ID,NULL AUTHOR_ID,NULL AUTHOR_ID,NULL AUTHOR_ID,NULL AUTHOR_ID,Guangde Li,An Ding,James B. Rottman,Yashvi Bhavsar,Clare Purcell,Rachit Jain,Ryan Alberry,NULL AUTHOR_ID,NULL AUTHOR_ID,NULL AUTHOR_ID,NULL AUTHOR_ID,Michael C. Holmes,NULL AUTHOR_ID,William Querbes,Ian E. Alexander,William E. Salomon
摘要
Recombinant adeno-associated virus (rAAV) vector gene delivery systems have demonstrated great promise in clinical trials but continue to face durability and dose-related challenges. Unlike rAAV gene therapy, integrating gene addition approaches can provide curative expression in mitotically active cells and pediatric populations. We explored a novel in vivo delivery approach based on an engineered transposase, Sleeping Beauty (SB100X), delivered as an mRNA within a lipid nanoparticle (LNP), in combination with an rAAV-delivered transposable transgene. This combinatorial approach achieved correction of ornithine transcarbamylase deficiency in the neonatal Spf
嘉心糖
薰硝壤
Emma
彭于彦祖
随机子
hl
YL
8R60d8
tianzml0
子车茗
梁朝伟
Singularity
shinysparrow
HEIKU
寻道图强
陈军
嗯哼
坚强的广山
tuanheqi
高大凌寒
皖公网安备34019202002308
