体内选择基因修正的 HSPC 可推进罕见干细胞疾病的基因治疗。

In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease.

机构信息

San Raffaele Telethon Institute for Gene Therapy, Milan, Italy; Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

San Raffaele Telethon Institute for Gene Therapy, Milan, Italy; Vita Salute San Raffaele University, Milan, Italy.

出版信息

Cell Stem Cell. 2019 Nov 7;25(5):592-593. doi: 10.1016/j.stem.2019.10.004.

DOI:10.1016/j.stem.2019.10.004

PMID:31703769

Abstract

Two recent papers (one by Román-Rodríguez et al., 2019 in this issue of Cell Stem Cell) highlight how the power of biological selection on hematopoietic stem cell fitness can facilitate gene therapies for Fanconi Anemia. A clinical trial using lentiviral gene replacement and a proof-of-concept targeted genome editing study show robust engraftment and expansion of gene-corrected cells at levels reaching therapeutic relevance.

摘要

两篇近期的论文（其中一篇由 Román-Rodríguez 等人发表于本期的《Cell Stem Cell》）强调了造血干细胞适应性选择的力量如何促进范可尼贫血症的基因治疗。一项使用慢病毒基因替换的临床试验和一项靶向基因组编辑的概念验证研究表明，经过基因校正的细胞具有强大的植入和扩增能力，其水平达到了治疗相关的要求。