AW-01. Transplantation of human hematopoietic stem cells with CCR5 knock out by CCR5-Uco-TALEN nuclease to NBSGW mice: pilot study

Cellular Therapy and Transplantation

article

Yaroslava V. Komarova¹  Alena I. Shakirova¹  Vladislav S. Sergeev¹  Svetlana A. Osipova²  Ekaterina V. Shchelina²  Anatoliya V. Onopchenko²  Marina L. Vasutina²  Olga G. Bredneva²  Kirill S. Yakovlev³  Yana R. Orshanskaya³  Konstantin V. Sivak³  Timofey E. Karpov¹  Albert R. Muslimov¹  Marina O. Popova¹  Kirill V. Lepik¹  Alexander D. Kulagin¹ 

[1] RM Gorbacheva Research Institute, Pavlov University;VA Almazov National Medical Research Centre;AA Smorodintsev Research Institute of Influenza

关键词: Transplantation;    hematopoietic stem cells;    genome editing;    TALEN;    CCR5;    model;    NBSGW mice;    NSG mice.;   

DOI  :  10.18620/ctt-1866-8836-2022-11-3-1-132

学科分类：肿瘤学

来源: Universitaetsklinikum Hamburg - Eppendorf / University Medical Center Hamburg - Eppendorf

【 摘 要 】

The method of hematopoietic stem cells (HSCs) transplantation to immunodeficient mice is an important component of the pre-clinical development of medicinal cell products. The traditional in vivo model used to study human hematopoiesis is NSG mice line, but currently the NBSGW is gaining interest [Adigbli, 2021]. Unlike NSG, NBSGW mice do not require pre-transplant conditioning due to the presence of a mutation of c-Kit, the SCF receptor, providing an advantage for transplanted cells. In the current study, for the first time in Russia, NBSGW line was used to study the engraftment and biodistribution of modified human HSCs after in vitro editing of the CCR5 gene mediated by TALEN nuclease. Our objective was to assess the engraftment and biodistribution after transplantation of hHSCs with CCR5 knockout mediated by TALEN nuclease.

【 授权许可】

CC BY