日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Cytosine base editing of LPA in transgenic mice averts large deletions.

对转基因小鼠中LPA的胞嘧啶碱基进行编辑可以避免大片段缺失。

Chuecos Marcel A, Park So Hyun, Bhakta Madhvi M, Too-Chiobi Usosa, Betancourth Daniel, Cao Mingming, De Giorgi Marco, Walkey Christopher J, Tiwari Anjana, Godin Biana, Assini Julia M, Palmer Donna J, Ng Philip, Boffa Michael B, Koschinsky Marlys L, Bao Gang, Lagor William R

A comprehensive atlas of AAV tropism in the mouse

小鼠体内AAV嗜性的综合图谱

Christopher J Walkey ,Kathy J Snow ,Jote Bulcha ,Aaron R Cox ,Alexa E Martinez ,M Cecilia Ljungberg ,Denise G Lanza ,Marco De Giorgi ,Marcel A Chuecos ,Michele Alves-Bezerra ,Carlos Flores Suarez ,Sean M Hartig ,Susan G Hilsenbeck ,Chih-Wei Hsu ,Ethan Saville ,Yaned Gaitan ,Jeff Duryea Jr ,Seth Hannigan ,Mary E Dickinson ,Oleg Mirochnitchenko ,Dan Wang ,Cathleen M Lutz ,Jason D Heaney ,Guangping Gao ,Stephen A Murray ,William R Lagor

Repair Drive improves gene editing in the liver

Repair Drive 改善肝脏基因编辑

De Giorgi, Marco; Park, So Hyun; Bao, Gang; Lagor, William R

Simple improvements in vector design afford substantial gains in AAV delivery of aggregation-slowing Aβ variants

载体设计的简单改进可以显著提高AAV递送减缓聚集的Aβ变体的效率。

Borgenheimer, Ella; Trueblood, Cameron; Nguyen, Bryan L; Lagor, William R; Jankowsky, Joanna L

Less is more: Allele-specific removal of dysfunctional RYR1 channel subunits

少即是多:等位基因特异性去除功能异常的RYR1通道亚基

Sun, Derek; Lagor, William R

Liver directed adeno-associated viral vectors to treat metabolic disease

靶向肝脏的腺相关病毒载体用于治疗代谢性疾病

Chuecos, Marcel A; Lagor, William R

Long-term efficacy and safety of cardiac genome editing for catecholaminergic polymorphic ventricular tachycardia

心脏基因组编辑治疗儿茶酚胺敏感性多形性室性心动过速的长期疗效和安全性

Moore, Oliver M; Aguilar-Sanchez, Yuriana; Lahiri, Satadru K; Hulsurkar, Mohit M; Alberto Navarro-Garcia, J; Word, Tarah A; Keefe, Joshua A; Barazi, Dean; Munivez, Elda M; Moore, Charles T; Parthasarathy, Vaidya; Davidson, Jaysón; Lagor, William R; Park, So Hyun; Bao, Gang; Miyake, Christina Y; Wehrens, Xander H T

Rescue of glutaric aciduria type I in mice by liver-directed therapies

通过肝脏靶向疗法挽救小鼠戊二酸尿症 I 型

Mercedes Barzi ,Collin G Johnson ,Tong Chen ,Ramona M Rodriguiz ,Madeline Hemmingsen ,Trevor J Gonzalez ,Alan Rosales ,James Beasley ,Cheryl K Peck ,Yunhan Ma ,Ashlee R Stiles ,Timothy C Wood ,Raquel Maeso-Diaz ,Anna Mae Diehl ,Sarah P Young ,Jeffrey I Everitt ,William C Wetsel ,William R Lagor ,Beatrice Bissig-Choisat ,Aravind Asokan ,Areeg El-Gharbawy ,Karl-Dimiter Bissig

Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier

通过设计能够穿过血脑屏障的衣壳受体相互作用,将 AAV 载体靶向中枢神经系统

Qin Huang, Albert T Chen, Ken Y Chan, Hikari Sorensen, Andrew J Barry, Bahar Azari, Qingxia Zheng, Thomas Beddow, Binhui Zhao, Isabelle G Tobey, Cynthia Moncada-Reid, Fatma-Elzahraa Eid, Christopher J Walkey, M Cecilia Ljungberg, William R Lagor, Jason D Heaney, Yujia A Chan, Benjamin E Deverman

Speg interactions that regulate the stability of excitation-contraction coupling protein complexes in triads and dyads

调节三联体和二联体中兴奋-收缩偶联蛋白复合物稳定性的 Speg 相互作用

Chang Seok Lee #, Sung Yun Jung #, Rachel Sue Zhen Yee, Nadia H Agha, Jin Hong, Ting Chang, Lyle W Babcock, Jorie D Fleischman, Benjamin Clayton, Amy D Hanna, Christopher S Ward, Denise Lanza, Ayrea E Hurley, Pumin Zhang, Xander H T Wehrens, William R Lagor, George G Rodney, Susan L Hamilton