Lessons learnt, and still to learn, in first in human stem cell trials.
Lessons learnt, and still to learn, in first in human stem cell trials.
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DOI：
10.1016/j.stemcr.2022.11.019
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发表时间：
2023-08-08
影响因子：
5.9
通讯作者：
Song, Jihwan
中科院分区：
文献类型：
--
作者：
Barker, Roger A.;Carpenter, Melissa;Jamieson, Catriona H. M.;Murry, Charles E.;Pellegrini, Graziella;Rao, Rajesh C.;Song, Jihwan
开发细胞疗法并不简单。本展望总结了一组干细胞学术研究者在不同临床领域工作的经验，旨在分享我们希望在开始之前知道的见解。这些包括(1)选择干细胞系和评估起始和最终产品的基因组，(2)熟悉GMP生产，试剂验证和供应链管理，(3)产品交付问题和额外的监管挑战，(4)临床试验设计和临床前研究之间的关系，以及(5)市场批准要求，途径和所需的合作伙伴关系。
Developing cellular therapies is not straightforward. This Perspective summarizes the experience of a group of academic stem cell investigators working in different clinical areas and aims to share insight into what we wished we knew before starting. These include (1) choosing the stem cell line and assessing the genome of both the starting and final product, (2) familiarity with GMP manufacturing, reagent validation, and supply chain management, (3) product delivery issues and the additional regulatory challenges, (4) the relationship between clinical trial design and preclinical studies, and (5) the market approval requirements, pathways, and partnerships needed.
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影响因子：
4.6
作者：
Popp B;Krumbiegel M;Grosch J;Sommer A;Uebe S;Kohl Z;Plötz S;Farrell M;Trautmann U;Kraus C;Ekici AB;Asadollahi R;Regensburger M;Günther K;Rauch A;Edenhofer F;Winkler J;Winner B;Reis A
通讯作者：
Reis A
影响因子：
64.8
作者：
Kriks, Sonja;Shim, Jae-Won;Piao, Jinghua;Ganat, Yosif M.;Wakeman, Dustin R.;Xie, Zhong;Carrillo-Reid, Luis;Auyeung, Gordon;Antonacci, Chris;Buch, Amanda;Yang, Lichuan;Beal, M. Flint;Surmeier, D. James;Kordower, Jeffrey H.;Tabar, Viviane;Studer, Lorenz
通讯作者：
Studer, Lorenz
预测标记指导分化以改善基于hESC的帕金森氏病疗法的临床翻译中的移植结果。
影响因子：
23.9
作者：
Kirkeby A;Nolbrant S;Tiklova K;Heuer A;Kee N;Cardoso T;Ottosson DR;Lelos MJ;Rifes P;Dunnett SB;Grealish S;Perlmann T;Parmar M
通讯作者：
Parmar M
影响因子：
4.5
作者：
Nittala, Muneeswar G.;Uji, Akihito;Sadda, Srinivas R.
通讯作者：
Sadda, Srinivas R.
影响因子：
8.8
作者：
Jiang Q;Isquith J;Ladel L;Mark A;Holm F;Mason C;He Y;Mondala P;Oliver I;Pham J;Ma W;Reynoso E;Ali S;Morris IJ;Diep R;Nasamran C;Xu G;Sasik R;Rosenthal SB;Birmingham A;Coso S;Pineda G;Crews L;Donohoe ME;Venter JC;Whisenant T;Mesa RA;Alexandrov LB;Fisch KM;Jamieson C
通讯作者：
Jamieson C