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

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

利用患者特异性体内基因编辑技术治疗罕见遗传疾病

Musunuru, Kiran; Grandinette, Sarah A; Wang, Xiao; Hudson, Taylor R; Briseno, Kevin; Berry, Anne Marie; Hacker, Julia L; Hsu, Alvin; Silverstein, Rachel A; Hille, Logan T; Ogul, Aysel N; Robinson-Garvin, Nancy A; Small, Juliana C; McCague, Sarah; Burke, Samantha M; Wright, Christina M; Bick, Sarah; Indurthi, Venkata; Sharma, Shweta; Jepperson, Michael; Vakulskas, Christopher A; Collingwood, Michael; Keogh, Katie; Jacobi, Ashley; Sturgeon, Morgan; Brommel, Christian; Schmaljohn, Ellen; Kurgan, Gavin; Osborne, Thomas; Zhang, He; Kinney, Kyle; Rettig, Garrett; Barbosa, Christopher J; Semple, Sean C; Tam, Ying K; Lutz, Cathleen; George, Lindsey A; Kleinstiver, Benjamin P; Liu, David R; Ng, Kim; Kassim, Sadik H; Giannikopoulos, Petros; Alameh, Mohamad-Gabriel; Urnov, Fyodor D; Ahrens-Nicklas, Rebecca C

Making the impossible possible: Baby KJ and the road map to personalized gene-editing care

化不可能为可能:KJ宝宝与个性化基因编辑护理路线图

Almendro, Vanessa; Kassim, Sadik H

The comparability tales: A phase-appropriate roadmap for CGT drug product development

可比性故事:CGT药物产品开发的阶段性路线图

Clément, Nathalie; Kassim, Sadik H; Leblanc, Daniel; Spink, Katy; Tomtishen, John

Single-cell multiplexed cytokine profiling of CD19 CAR-T cells reveals a diverse landscape of polyfunctional antigen-specific response

CD19 CAR-T 细胞的单细胞多重细胞因子分析揭示了多功能抗原特异性反应的多样化格局

Qiong Xue, Emily Bettini, Patrick Paczkowski, Colin Ng, Alaina Kaiser, Timothy McConnell, Olja Kodrasi, Máire F Quigley, James Heath, Rong Fan, Sean Mackay, Mark E Dudley, Sadik H Kassim, Jing Zhou

Chemotherapy-refractory diffuse large B-cell lymphoma and indolent B-cell malignancies can be effectively treated with autologous T cells expressing an anti-CD19 chimeric antigen receptor

化疗难治性弥漫性大B细胞淋巴瘤和惰性B细胞恶性肿瘤可采用表达抗CD19嵌合抗原受体的自体T细胞进行有效治疗。

Kochenderfer, James N; Dudley, Mark E; Kassim, Sadik H; Somerville, Robert P T; Carpenter, Robert O; Stetler-Stevenson, Maryalice; Yang, James C; Phan, Giao Q; Hughes, Marybeth S; Sherry, Richard M; Raffeld, Mark; Feldman, Steven; Lu, Lily; Li, Yong F; Ngo, Lien T; Goy, Andre; Feldman, Tatyana; Spaner, David E; Wang, Michael L; Chen, Clara C; Kranick, Sarah M; Nath, Avindra; Nathan, Debbie-Ann N; Morton, Kathleen E; Toomey, Mary Ann; Rosenberg, Steven A

A pilot trial using lymphocytes genetically engineered with an NY-ESO-1-reactive T-cell receptor: long-term follow-up and correlates with response

一项使用经基因工程改造、表达NY-ESO-1反应性T细胞受体的淋巴细胞的试点试验:长期随访及疗效相关性分析

Robbins, Paul F; Kassim, Sadik H; Tran, Thai L N; Crystal, Jessica S; Morgan, Richard A; Feldman, Steven A; Yang, James C; Dudley, Mark E; Wunderlich, John R; Sherry, Richard M; Kammula, Udai S; Hughes, Marybeth S; Restifo, Nicholas P; Raffeld, Mark; Lee, Chyi-Chia R; Li, Yong F; El-Gamil, Mona; Rosenberg, Steven A

Tumor-infiltrating lymphocytes genetically engineered with an inducible gene encoding interleukin-12 for the immunotherapy of metastatic melanoma

利用可诱导的白细胞介素-12 基因工程改造肿瘤浸润淋巴细胞,用于转移性黑色素瘤的免疫治疗

Ling Zhang, Richard A Morgan, Joal D Beane, Zhili Zheng, Mark E Dudley, Sadik H Kassim, Azam V Nahvi, Lien T Ngo, Richard M Sherry, Giao Q Phan, Marybeth S Hughes, Udai S Kammula, Steven A Feldman, Mary Ann Toomey, Sid P Kerkar, Nicholas P Restifo, James C Yang, Steven A Rosenberg

Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia

腺相关病毒8型基因疗法可显著降低纯合子和杂合子家族性高胆固醇血症人源化小鼠模型的血浆胆固醇水平。

Kassim, Sadik H; Li, Hui; Bell, Peter; Somanathan, Suryanarayan; Lagor, William; Jacobs, Frank; Billheimer, Jeffrey; Wilson, James M; Rader, Daniel J

From lipoproteins to chondrocytes: a brief summary of the European Medicines Agency's regulatory guidelines for advanced therapy medicinal products

从脂蛋白到软骨细胞:欧洲药品管理局高级疗法药品监管指南简要概述

Kassim, Sadik H; Somerville, Robert P T

Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia

在纯合子家族性高胆固醇血症小鼠模型中表达人低密度脂蛋白受体的AAV8载体的生物分布

Chen, Shu-Jen; Sanmiguel, Julio; Lock, Martin; McMenamin, Deirdre; Draper, Christine; Limberis, Maria P; Kassim, Sadik H; Somanathan, Suryanarayan; Bell, Peter; Johnston, Julie C; Rader, Daniel J; Wilson, James M