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

Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.

在小鼠模型中对 CRISPR-Cas9、慢病毒转导和碱基编辑治疗镰状细胞病进行比较分析。

Butt Henna, Sathish Shruti, London Evan, Le Anh, Li Quan, Gudmundsdottir Bjorg, Lee Duck-Yeon, Burke Emma V, Yates Bradley P, Liu David R, Hsieh Matthew, Leonard Alexis, Eaton William A, Uchida Naoya, Pierciey Francis J Jr, Newby Gregory A, Tisdale John F, Demirci Selami

Mitochondrial DNA copy number reduction via in vitro TFAM knockout remodels the nuclear epigenome and transcriptome

体外TFAM敲除导致线粒体DNA拷贝数减少,进而重塑核表观基因组和转录组。

Win, Phyo W; Nguyen, Julia; Shin, Elly H; Nagano, Tyler S; Selimi, Brent; Hong, Katie; Meybodi, Anahita M; Yates, Bradley P; Burke, Emma V; Carter, David E; Newby, Gregory A; Newcomb, Charles; Arking, Dan E; Castellani, Christina A

Author Correction: In vivo base editing extends lifespan of a humanized mouse model of prion disease

作者更正:体内碱基编辑延长了朊病毒病人源化小鼠模型的寿命

An, Meirui; Davis, Jessie R; Levy, Jonathan M; Serack, Fiona E; Harvey, John W; Brauer, Pamela P; Pirtle, Catherine P; Berríos, Kiara N; Newby, Gregory A; Yeh, Wei-Hsi; Kamath, Nikita; Mortberg, Meredith; Lian, Yuan; Howard, Michael; DeSouza-Lenz, Kendrick; Guzman, Kenia; Thai, Aaron; Graffam, Samantha; Laversenne, Vanessa; Coffey, Alissa A; Frei, Jeannine; Pierce, Sarah E; Safar, Jiri G; Deverman, Benjamin E; Minikel, Eric Vallabh; Vallabh, Sonia M; Liu, David R

In vivo base editing extends lifespan of a humanized mouse model of prion disease.

体内碱基编辑延长了朊病毒疾病人源化小鼠模型的寿命

An Meirui, Davis Jessie R, Levy Jonathan M, Serack Fiona E, Harvey John W, Brauer Pamela P, Pirtle Catherine P, Berríos Kiara N, Newby Gregory A, Yeh Wei-Hsi, Kamath Nikita, Mortberg Meredith, Lian Yuan, Howard Michael, DeSouza-Lenz Kendrick, Guzman Kenia, Thai Aaron, Graffam Samantha, Laversenne Vanessa, Coffey Alissa A, Frei Jeannine, Pierce Sarah E, Safar Jiri G, Deverman Benjamin E, Minikel Eric Vallabh, Vallabh Sonia M, Liu David R

Precisely defining disease variant effects in CRISPR-edited single cells

精确定义 CRISPR 编辑的单细胞中的疾病变异效应

Baglaenko, Yuriy; Mu, Zepeng; Curtis, Michelle; Mire, Hafsa M; Jayanthi, Vidyashree; Al Suqri, Majd; Liu, Cassidy; Agnew, Ryan; Nathan, Aparna; Mah-Som, Annelise Yoo; Liu, David R; Newby, Gregory A; Raychaudhuri, Soumya

Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells

系统优化主编辑策略,以实现人呼吸道上皮细胞中CFTR F508del突变的有效功能性矫正

Sousa, Alexander A; Hemez, Colin; Lei, Lei; Traore, Soumba; Kulhankova, Katarina; Newby, Gregory A; Doman, Jordan L; Oye, Keyede; Pandey, Smriti; Karp, Philip H; McCray, Paul B Jr; Liu, David R

GFP-on mouse model for interrogation of in vivo gene editing.

用于研究体内基因编辑的 GFP 标记小鼠模型

Dib Carla, Queenan Jack A, Swartzrock Leah, Willner Hana, Denis Morgane, Ahmed Nouraiz, Moulana Zada Fareha, Borges Beltran, Charlesworth Carsten T, Lum Tony, Yates Bradley P, Kwon Caleb Y, Scorzo Augustino V, Davis Scott C, Davis Jessie R, He Ran, Xie Jun, Gao Guangping, MacKenzie Tippi C, Liu David R, Newby Gregory A, Czechowicz Agnieszka D

Engraftment and persistence of HBB base-edited hematopoietic stem cells in nonhuman primates

HBB碱基编辑的造血干细胞在非人灵长类动物中的植入和持久性

Radtke, Stefan; Fields, Emily; Swing, Kyle; Kanestrom, Greta; Yen, Jonathan S; Pande, Dnyanada; Enstrom, Mark R; Humbert, Olivier; Weiss, Mitchell J; Liu, David R; Newby, Gregory A; Kiem, Hans-Peter

Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis

碱基编辑和纳米颗粒转染气道细胞类型对于治疗囊性纤维化至关重要

Kavanagh, Erin W; Joynt, Anya T; Pion, Audrey R; Eastman, Alice C; Parr, Alianna I; Starego, Katherine L; Jain, Manav; Shannon, Sydney R; Yoo, Edwin; Newby, Gregory A; Tzeng, Stephany Y; Sharma, Neeraj; Green, Jordan J; Cutting, Garry R

Amphiphilic shuttle peptide delivers base editor ribonucleoprotein to correct the CFTR R553X mutation in well-differentiated airway epithelial cells.

两亲性穿梭肽将碱基编辑器核糖核蛋白递送至分化良好的气道上皮细胞,以纠正 CFTR R553X 突变

Kulhankova Katarina, Cheng Anna X, Traore Soumba, Auger Maud, Pelletier Mia, Hervault Maxime, Wells Kevin D, Green Jonathan A, Byrne Addison, Nelson Benjamin, Sponchiado Mariana, Boosani Chandra, Heffner Caleb S, Snow Kathy J, Murray Stephen A, Villacreses Raul A, Rector Michael V, Gansemer Nicholas D, Stoltz David A, Allamargot Chantal, Couture Frédéric, Hemez Colin, Hallée Stéphanie, Barbeau Xavier, Harvey Mario, Lauvaux Coraline, Gaillet Bruno, Newby Gregory A, Liu David R, McCray Paul B Jr, Guay David