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

Novel humanized loss-of-function NF1 mouse model of juvenile myelomonocytic leukemia.

新型人源化功能缺失型 NF1 小鼠幼年型骨髓单核细胞白血病模型。

Sinha Roshani, Patil Rachana Vinay, Romano Rosa, Sharma Devesh, Lee Esmond, Perriman Rhonda, Takeda Saori, Lesch Benjamin J, Yao Zhenyu, Liu Y Lucy, Cromer M Kyle, Porteus Matthew H, Bertaina Alice

Rewiring endogenous genes in CAR T cells for tumour-restricted payload delivery

通过重编程CAR T细胞中的内源基因,实现肿瘤特异性有效载荷递送。

Chen, Amanda X Y; Yap, Kah Min; Kim, Joelle S; Sek, Kevin; Huang, Yu-Kuan; Dunbar, Phoebe A; Wiebking, Volker; Armitage, Jesse D; Munoz, Isabelle; Todd, Kirsten L; Derrick, Emily B; Nguyen, Dat; Tong, Junming; Chan, Cheok Weng; Hoang, Thang X; Audsley, Katherine M; van Elsas, Marit J; Middelburg, Jim; Lee, Joel N; de Menezes, Maria N; Cole, Thomas J; Li, Jasmine; Scheffler, Christina; Scott, Andrew M; Mackay, Laura K; Waithman, Jason; Oliaro, Jane; Harrison, Simon J; Parish, Ian A; Lai, Junyun; Porteus, Matthew H; House, Imran G; Darcy, Phillip K; Beavis, Paul A

A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expression.

差异化的β-珠蛋白基因替换策略利用异源内含子来恢复生理表达

Wallace Kirby A, Gerstenberg Trevor L, Ennis Craig L, Perez-Bermejo Juan A, Partridge James R, Bandoro Christopher, Matern William M, Andreoletti Gaia, Krassovsky Kristina, Kabir Shaheen, Lalisan Cassandra D, Churi Aishwarya R, Chew Glen M, Corbo Lana, Vincelette Jon E, Klasson Timothy D, Silva Brian J, Strukov Yuri G, Quejarro B Joy, Hill Kaisle A, Treusch Sebastian, Grogan Jane L, Dever Daniel P, Porteus Matthew H, Wienert Beeke

Discovery of a pre-vein progenitor that requires VEGF/ERK inhibition to complete vein differentiation.

发现一种前静脉祖细胞,需要抑制 VEGF/ERK 才能完成静脉分化。

Ang Lay Teng, Zheng Sherry Li, Liu Kevin J, Masaltseva Anastasiia, Winters June, von Creytz Isabel, Jha Sawan K, Yin Qingqing, Qian Crystal, Xiong Xiaochen, Dailamy Amir, Xi Ellie, Alcocer Juan C, Sorensen Daniel W, She Richard, Smolyar Karina, Szumska Dorota, Nornes Svanhild, Martin Renata M, Lesch Benjamin J, Restrepo Nicole K, Sun Wenfei, Weissman Jonathan S, Lickert Heiko, Porteus Matthew H, Skylar-Scott Mark A, Mosimann Christian, Sumanas Saulius, Val Sarah De, Prescott Joseph B, Red-Horse Kristy, Loh Kyle M

Durable reconstitution of sinonasal epithelium by transplant of CFTR gene corrected airway stem cells.

通过移植 CFTR 基因校正的气道干细胞,实现鼻窦上皮的持久重建

Bravo Dawn T, Vaidyanathan Sriram, Baker Jeannette, Sinha Vrishti, Tsai Esmond, Roozdar Pooya, Kong William W, Atkinson Patrick J, Patel Zara M, Hwang Peter H, Rao Vidya K, Negrin Robert S, Wine Jeffrey J, Milla Carlos, Sellers Zachary M, Desai Tushar J, Porteus Matthew H, Nayak Jayakar V

Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells.

通过 Cas9 介导将天然变异体插入造血干细胞和祖细胞,增强红细胞生成输出

Luna Sofia E, Camarena Joab, Hampton Jessica P, Majeti Kiran R, Charlesworth Carsten T, Soupene Eric, Selvaraj Sridhar, Jia Kun, Sheehan Vivien A, Cromer M Kyle, Porteus Matthew H

Failure of metabolic checkpoint control during late-stage granulopoiesis drives neutropenia in reticular dysgenesis

晚期粒细胞生成过程中代谢检查点控制的失败导致网状细胞发育不全患者出现中性粒细胞减少症。

Wang, Wenqing; Arreola, Martin; Mathews, Thomas; DeVilbiss, Andrew; Zhao, Zhiyu; Martin-Sandoval, Misty; Mohammed, Abdulvasey; Benegiamo, Giorgia; Awani, Avni; Goeminne, Ludger; Dever, Daniel; Nakauchi, Yusuke; Porteus, Matthew H; Pavel-Dinu, Mara; Al-Herz, Waleed; Auwerx, Johan; Morrison, Sean J; Weinacht, Katja G

Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

基因校正的RAG2-SCID人类造血干细胞可恢复V(D)J重组酶并挽救淋巴细胞缺陷

Pavel-Dinu, Mara; Gardner, Cameron L; Nakauchi, Yusuke; Kawai, Tomoki; Delmonte, Ottavia M; Palterer, Boaz; Bosticardo, Marita; Pala, Francesca; Viel, Sebastien; Malech, Harry L; Ghanim, Hana Y; Bode, Nicole M; Kurgan, Gavin L; Detweiler, Angela M; Vakulskas, Christopher A; Neff, Norma F; Sheikali, Adam; Menezes, Sherah T; Chrobok, Jade; Hernández González, Elaine M; Majeti, Ravindra; Notarangelo, Luigi D; Porteus, Matthew H

A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System

治疗造血缺陷的DNA密码:血液和免疫系统单基因疾病的新型细胞疗法

Porteus, Matthew H; Pavel-Dinu, Mara; Pai, Sung-Yun

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

增强同源定向修复以实现造血干/祖细胞中高效的基因编辑

De Ravin, Suk See; Brault, Julie; Meis, Ronald J; Liu, Siyuan; Li, Linhong; Pavel-Dinu, Mara; Lazzarotto, Cicera R; Liu, Taylor; Koontz, Sherry M; Choi, Uimook; Sweeney, Colin L; Theobald, Narda; Lee, GaHyun; Clark, Aaron B; Burkett, Sandra S; Kleinstiver, Benjamin P; Porteus, Matthew H; Tsai, Shengdar; Kuhns, Douglas B; Dahl, Gary A; Headey, Stephen; Wu, Xiaolin; Malech, Harry L