mRNA-based enzyme replacement for propionic acidaemia

doi:10.1038/s41574-025-01150-8

2025-07-07

PRE

Chuang LIU *

DOI：10.1038/s41574-025-01150-8

原文链接

原文求助

摘要

期刊

Nature Reviews Endocrinology

IF:

论文数:

1.0W

被引数:

10.5W

机构

brigham and women’s hospital

学者数:

1.2K

论文数: 510

被引数: 0

引用论文

Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia 丙酸血症的首次人类1/2期mRNA试验的中期分析

Nature

IF0

2024-04-03

OAAI

Dwight Koeberl; Andreas Schulze; Neal Sondheimer; Gerald S. Lipshutz; Tarekegn Geberhiwot; Lerong Li; Rajnish Saini; Junxiang Luo; Vanja Sikirica; Ling Jin; Min Liang; Mary Leuchars; Stephanie Grunewald

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease 针对罕见遗传病的患者特异性体内基因编辑治疗

New England Journal of Medicine

IF0

2025-06-12

PREAI

Kiran Musunuru; Sarah A. Grandinette; Xiao Wang; Taylor R. Hudson; Kevin Briseno; Anne Marie Berry; Julia L. Hacker; Alvin Hsu; Rachel A. Silverstein; Logan T. Hille; Aysel N. Ogul; Nancy A. Robinson-Garvin; Juliana C. Small; Sarah McCague; Samantha M. Burke; Christina M. Wright; Sarah Bick; Venkata Indurthi; Shweta Sharma; Michael Jepperson; Christopher A. Vakulskas; Michael Collingwood; Katie Keogh; Ashley Jacobi; Morgan Sturgeon; Christian Brommel; Ellen Schmaljohn; Gavin Kurgan; Thomas Osborne; He Zhang; Kyle Kinney; Garrett Rettig; Christopher J. Barbosa; Sean C. Semple; Ying K. Tam; Cathleen Lutz; Lindsey A. George; Benjamin P. Kleinstiver; David R. Liu; Kim Ng; Sadik H. Kassim; Petros Giannikopoulos; Mohamad-Gabriel Alameh; Fyodor D. Urnov; Rebecca C. Ahrens-Nicklas