The bleeding edge: broadening horizons for hematopoietic stem cell therapies.

IF 12.8 1区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Trends in molecular medicine Pub Date : 2025-03-21 DOI:10.1016/j.molmed.2025.03.003

Spencer D Shelton, Vijay G Sankaran

引用次数: 0

Abstract

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.

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来源期刊

Trends in molecular medicine 医学-生化与分子生物学

CiteScore

24.60

自引率

0.00%

发文量

142

审稿时长

6-12 weeks

期刊介绍： Trends in Molecular Medicine (TMM) aims to offer concise and contextualized perspectives on the latest research advancing biomedical science toward better diagnosis, treatment, and prevention of human diseases. It focuses on research at the intersection of basic biology and clinical research, covering new concepts in human biology and pathology with clear implications for diagnostics and therapy. TMM reviews bridge the gap between bench and bedside, discussing research from preclinical studies to patient-enrolled trials. The major themes include disease mechanisms, tools and technologies, diagnostics, and therapeutics, with a preference for articles relevant to multiple themes. TMM serves as a platform for discussion, pushing traditional boundaries and fostering collaboration between scientists and clinicians. The journal seeks to publish provocative and authoritative articles that are also accessible to a broad audience, inspiring new directions in molecular medicine to enhance human health.