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

Hematopoietic Stem-Cell Gene Therapy for Cystinosis

胱氨酸病的造血干细胞基因治疗

Barshop, Bruce A; Ball, Edward D; Benador, Nadine; Trauner, Doris; Phillips, Susan; Dohil, Ranjan; Afshari, Natalie A; Roy, Sohini; Campo Fernandes, Beatriz; Kohn, Donald; Shayan, Katayoon; Everett, John K; Bushman, Frederic D; Midgley, Julian; Liang, Hong; Sawyers, Anne; Gangoiti, Jon A; Panchal, Maulik; Ahmed, Imama; Cherqui, Stephanie

CD4(+) T cells mediate CAR-T cell-associated immune-related adverse events after BCMA CAR-T cell therapy.

CD4(+) T 细胞介导 BCMA CAR-T 细胞治疗后 CAR-T 细胞相关的免疫相关不良事件。

Ho Matthew, Paruzzo Luca, Noll Julia Han, Stella Federico, Devi Pooja, Ndeupen Sonia, Day Yael A, Chen Gregory M, Cohen Ivan J, Ramirez-Fernandez Angel, Waxman Adam, Kapur Shivani, Chen Fang, Xu Rong, Huff Andrew, Jarocha Danuta, Patel Vrutti, Bochi-Layec Audrey C, Ramasubramanian Ranjani, Liu Shan, Bouvier Riemke, de Souza Vitor B, Patel Heta, Li Ziyu, Carturan Alberto, Michener Peter, Hopkins Caitlin R, Koucky Owen, Minehart Janna, Dimitri Alex, Nabar Neel R, Hasanali Zainul S, Ciccarelli Bryan T, Hung Putzer, Williams Erik, Bartoszek Robert, Lavorando Maya, Mohan Suyash, Gonzalez Vanessa E, Porazzi Patrizia, Bhoj Vijay G, Apostolidis Sokratis A, Vogl Dan T, Porter David L, Scholler John, Diorio Caroline, Roche Aoife M, Everett John K, Bushman Frederic D, Nathanson Katherine L, Stadtmauer Edward A, Susanibar-Adaniya Sandra P, Garfall Alfred L, Ruella Marco, Cohen Adam D, Fraietta Joseph A

CD4+ T-Cell Lymphoma Harboring a Chimeric Antigen Receptor Integration in TP53

CD4+ T 细胞淋巴瘤携带嵌合抗原受体整合于 TP53

Perica, Karlo; Jain, Nayan; Scordo, Michael; Patel, Ruchi; Eren, Ozgur Can; Patel, Utsav; Gundem, Gunes; Domenico, Dylan; Mitra, Sneha; Socci, Nicholas D; Everett, John K; Roche, Aoife M; Petrichenko, Angelina; Shah, Gunjan L; Arcila, Maria E; Borsu, Laetitia; Park, Jae H; Horwitz, Steven M; Giralt, Sergio A; Dogan, Ahmet; Leslie, Christina; Papaemmanuil, Elli; Bushman, Frederic D; Usmani, Saad Z; Sadelain, Michel; Mailankody, Sham

Pooled CAR-T screening in pig-tailed macaques identifies designs with enhanced proliferation, trafficking, and persistence

在豚尾猕猴中进行的混合 CAR-T 筛选鉴定出具有增强的增殖、迁移和持久性的设计。

Maynard, Lucy H; Cavanaugh, Eric J; Zhu, Haiying; Starke, Carly E; Doherty, Sarah M; Einhaus, Teresa; Pérez-Osorio, Ailyn C; Stensland, Laurence; Blair, Cameron; Roche, Aoife M; Everett, John K; Murnane, Robert D; Hoffman, Michelle; Nelson, Veronica; Herrin, Sarah; Littlewood, Chad; Camou, Kaycee; Wilson, Erica; Wessel, Christopher; Bushman, Frederic D; Jerome, Keith R; Kiem, Hans-Peter; Peterson, Christopher W

Small molecule inhibition of SUMOylation increases expression from AAV vectors both during and after initial transduction in mice

小分子抑制剂SUMO化可增强小鼠体内AAV载体在初始转导期间及转导后的表达。

Seleme, Maria C; Kasimsetty, Aradhana; Hwang, Young; Lee, Carole; Roche, Aoife M; Henriksen, Allysen C; Everett, John K; Bushman, Frederic D; Sabatino, Denise E

Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies.

用于治疗肌营养不良症的 CGMP iPSC 衍生肌源性祖细胞产品的临床前质量、安全性和有效性

Azzag Karim, Magli Alessandro, Kiley James, Sumstad Darin, Kadidlo Diane, Crist Sarah B, Norris Beverly, Ahlquist Aaron, Hocum Stone Laura L, Everett John, Shappa Faustich Jill, Seelig Davis, Rangarajan Parthasarathy, Kim Hyunkee, Flory Craig, Bushman Frederic, Ramachandran Sabarinathan, Kang Peter B, Schumacher Robert J, Wagner John E, Kyba Michael, Graham Melanie L, McKenna David H, Perlingeiro Rita C R

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

利用靶向造血干细胞的脂质纳米颗粒构建致死性α-地中海贫血小鼠模型,并通过慢病毒基因治疗进行治疗。

Chappell, Maxwell E; Breda, Laura; Tricoli, Lucas; Guerra, Amaliris; Jarocha, Danuta; Castruccio Castracani, Carlo; Papp, Tyler E; Tanaka, Naoto; Hamilton, Nolan; Triebwasser, Michael P; Ghiaccio, Valentina; Fedorky, Megan T; Gollomp, Kandace L; Bochenek, Veronica; Roche, Aoife M; Everett, John K; Cook, Emma J; Bushman, Frederic D; Teawtrakul, Nattiya; Glentis, Stavros; Kattamis, Antonis; Mui, Barbara L; Tam, Ying K; Weissman, Drew; Abdulmalik, Osheiza; Parhiz, Hamideh; Rivella, Stefano

Modulation of AAV transduction and integration targeting by topoisomerase poisons

利用拓扑异构酶抑制剂调控AAV转导和整合靶向

Kasimsetty, Aradhana; Hwang, Young; Everett, John K; McFarland, Alexander G; Zolnoski, Sonja A; Lu, Tianyu; Roche, Aoife M; Martínez-García, Pedro Manuel; Sabatino, Denise E; Bushman, Frederic D

The HIV-1 Capsid-Targeted Inhibitor GSK878 Alters Selection of Target Sites for HIV DNA Integration

HIV-1衣壳靶向抑制剂GSK878改变了HIV DNA整合靶位点的选择

Marquis, Kaitlin A; Everett, John; Cantu, Adrian; McFarland, Alexander; Sherrill-Mix, Scott; Krystal, Mark; Parcella, Kyle; Gillis, Eric; Fridell, Robert A; Bushman, Frederic D

Chemotherapy-induced reversal of ciltacabtagene autoleucel-associated movement and neurocognitive toxicity

化疗诱导的西他卡巴他基因自体白细胞相关运动和神经认知毒性的逆转

Graham, Charlotte E; Lee, Won-Ho; Wiggin, Hadley R; Supper, Valentina M; Leick, Mark B; Birocchi, Filippo; Yee, Andrew J; Petrichenko, Angelina; Everett, John; Bushman, Frederic D; Sadrzadeh, Hossein; Rapalino, Otto; Chiu, Daniel; Arrillaga-Romany, Isabel; Maus, Marcela V; Frigault, Matthew J; Gallagher, Kathleen M E