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
热心市民小红花
Leonardi
嗯哼
wen
薰硝壤
夏来
shinysparrow
Singularity
敬老院N号
sissiarno
小蚂蚁
Dore
古的古的
yufanhui
敬老院1号
s615
rosalieshi
zeo
香芋
吴彦祖
皖公网安备34019202002308
