Hematopoietic stem cell gene therapy to halt neurodegeneration

被引：2

作者：

Biffi, Alessandra ^{[1
,2
]}

机构：

[1] Univ Padua, Dept Women & Childs Hlth, Div Pediat Hematol Oncol & Stem Cell Transplantat, Padua, Italy

[2] Padova Univ Hosp, Padua, Italy

来源：

NEUROTHERAPEUTICS | 2024年 / 21卷 / 04期

关键词：

Hematopoietic stem cells; Gene therapy; Microglia; CENTRAL-NERVOUS-SYSTEM; ONSET METACHROMATIC LEUKODYSTROPHY; MOUSE MODEL; OPEN-LABEL; MICROGLIA; REVEALS; HEALTH; TRANSPLANTATION; ENGRAFTMENT; CORRECTS;

D O I：

10.1016/j.neurot.2024.e00440

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Microglia play fundamental roles in multiple pathological primary and secondary processes affecting the central nervous system that ultimately result in neurodegeneration and for this reason they are considered as a key therapeutic target in several neurodegenerative diseases. Microglia-targeted therapies are directed at either restoring or modulating microglia function, to redirect their functional features toward neuroprotection. Among these strategies, hematopoietic stem cell gene therapy have proven to be endowed with a unique potential for replacing diseased microglia with engineered, transplant progeny cells that can integrate and exert relevant beneficial effects in the central nervous system of patients affected by inherited and acquired neurodegenerative conditions.

引用

页数：5

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