期刊
BONE MARROW TRANSPLANTATION
卷 54, 期 12, 页码 1940-1950出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/s41409-019-0510-8
关键词
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资金
- Jurgen Manchot Stiftung
- Fortune Tubingen [2412-0-0, 2485-0-0]
- University Children's Hospital of Tubingen
Allogeneic hematopoietic stem cell transplantation (HSCT) is a standard therapeutic intervention for hematological malignancies and several monogenic diseases. However, this approach has limitations related to lack of a suitable donor, graft-versus-host disease and infectious complications due to immune suppression. On the contrary, autologous HSCT diminishes the negative effects of allogeneic HSCT. Despite the good efficacy, earlier gene therapy trials with autologous HSCs and viral vectors have raised serious safety concerns. However, the CRISPR/Cas9-edited autologous HSCs have been proposed to be an alternative option with a high safety profile. In this review, we summarized the possibility of CRISPR/Cas9-mediated autologous HSCT as a potential treatment option for various diseases supported by preclinical gene-editing studies. Furthermore, we discussed future clinical perspectives and possible clinical grade improvements of CRISPR/cas9-mediated autologous HSCT.
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