前沿领域：拓展造血干细胞疗法的视野
The bleeding edge: broadening horizons for hematopoietic stem cell therapies.
作者信息
Shelton Spencer D, Sankaran Vijay G
机构信息
Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA; Howard Hughes Medical Institute, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA; Howard Hughes Medical Institute, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
出版信息
Trends Mol Med. 2025 Apr;31(4):301-303. doi: 10.1016/j.molmed.2025.03.003. Epub 2025 Mar 21.
Recent reports of gene therapy using autologous hematopoietic stem cell transplantation (HSCT) have addressed protein deficiencies of extra-hematopoietic origin. In a recent study, Srivastava et al. report that patients with hemophilia A receiving F8 lentiviral HSCT gene therapy achieved lasting factor VIII restoration and clinical improvement, marking an advance that could enable broader applications of HSCT.
近期有关使用自体造血干细胞移植（HSCT）进行基因治疗的报道涉及了造血外源性蛋白质缺陷。在最近一项研究中，斯里瓦斯塔瓦等人报告称，接受F8慢病毒HSCT基因治疗的A型血友病患者实现了持久的凝血因子VIII恢复和临床改善，这标志着一项进展，可能使HSCT得到更广泛应用。
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