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

Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy

镰状细胞病基因治疗:美国移植和细胞治疗学会以及国际细胞和基因治疗学会的实践建议

Sharma, Akshay; Kassim, Adetola; Thompson, Alexis; Williams, David A; Liu, Hien D; Boelens, Jaap J; LaBelle, James L; Tisdale, John; de la Fuente, Josu; Kanter, Julie; Krishnamurti, Lakshmanan; Pecker, Lydia H; Walters, Mark C; Eapen, Mary; Porteus, Matt; Bhatia, Monica; Soni, Sandeep; Corbacioglu, Selim; John, Tami D; Cancio, María I

Engineered hematopoietic stem cells give rise to therapeutic antibody secreting B cells

经基因工程改造的造血干细胞可产生分泌治疗性抗体的B细胞。

Porteus, Matthew; Luna, Sofia; Feist, William; Utz, Ashley; Afaghani, Jumana; Miyauchi, Masashi; Ghanim, Hana; Ekman, Freja; Amaya, Anais; Selvaraj, Sridhar; Russkamp, Norman; Schmiderer, Ludwig

Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial

使用抗CD117抗体治疗范可尼贫血的无辐射、无白消安干细胞移植:一项1b期试验

Agarwal, Rajni; Bertaina, Alice; Soco, Charmaine; Long-Boyle, Janel R; Saini, Gopin; Kunte, Nivedita; Hiroshima, Lyndsie; Chan, Yan Y; Willner, Hana; Krampf, Mark R; Nofal, Rofida; Barbarito, Giulia; Sen, Sushmita; Van Hentenryck, Maite; Walck, Emily; Scheck, Amelia; Perriman, Rhonda J; Bouge, Alisha; Istomina, Elena; Din, Hena Naz; Klinger, Edna F; Cheng, Jerry C; Wlodarski, Marcin W; Boelens, Jaap J; Shizuru, Judith A; Pang, Wendy W; Weinberg, Kenneth; Parkman, Robertson; Roncarolo, Maria Grazia; Porteus, Matthew; Czechowicz, Agnieszka

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

Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis

设计合成信号受体以实现无促红细胞生成素的红细胞生成

Aadit P Shah #, Kiran R Majeti #, Freja K Ekman, Sridhar Selvaraj, Devesh Sharma, Roshani Sinha, Eric Soupene, Prathamesh Chati, Sofia E Luna, Carsten T Charlesworth, Travis McCreary, Benjamin J Lesch, Tammy Tran, Simon N Chu, Matthew H Porteus, M Kyle Cromer1

Unveiling the cut-and-repair cycle of designer nucleases in human stem and T cells via CLEAR-time dPCR

利用 CLEAR-time dPCR 揭示人类干细胞和 T 细胞中设计核酸酶的切割和修复循环

Nathan White,John Alexander Chalk,Yi-Ting Hu,Samuel Mark Pins,Chinnu Rose Joseph,Panagiotis Antoniou,Sandra Wimberger,Stina Svensson,Soraia Patricia Caetano-Silva,Anne Charlotte Adriane Mudde,Rajeev Rai,Sridhar Selvaraj,William Nelson Feist,Marianna Romito,Grzegorz Sienski,Roberto Nitsch,Claire Booth,Giorgia Santilli,Alessia Cavazza,Matthew Hebden Porteus,Marcello Maresca,Adrian James Thrasher,Giandomenico Turchiano

Multilayered HIV-1 resistance in HSPCs through CCR5 Knockout and B cell secretion of HIV-inhibiting antibodies

通过CCR5敲除和B细胞分泌HIV抑制抗体,造血干细胞中形成多层HIV-1抵抗力

William N Feist # ,Sofia E Luna # ,Kaya Ben-Efraim ,Maria V Filsinger Interrante ,Alvaro Amorin ,Nicole M Johnston ,Theodora U J Bruun ,Ashley Utz ,Hana Y Ghanim ,Benjamin J Lesch ,Theresa M McLaughlin ,Amanda M Dudek ,Matthew H Porteus

Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases

克服罕见病和超罕见病基因和细胞疗法商业化前期可行性障碍

Barrett, David; Cannon, Paula M; Mingozzi, Federico; Porteus, Matthew; Rivière, Isabelle; Flotte, Terence R

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