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Review
. 2024 Jul;21(4):e00440.
doi: 10.1016/j.neurot.2024.e00440. Epub 2024 Sep 14.

Hematopoietic stem cell gene therapy to halt neurodegeneration

Alessandra Biffi  1
Affiliations
Review

Hematopoietic stem cell gene therapy to halt neurodegeneration

Alessandra Biffi. Neurotherapeutics. 2024 Jul.

Abstract

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.

Keywords: Gene therapy; Hematopoietic stem cells; Microglia.

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Conflict of interest statement

Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Alessandra Biffi reports a relationship with Orchard Therapeutics that includes: consulting or advisory and funding grants. If there are other authors, they declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Figures

Fig. 1
Fig. 1
Mechanisms of benefit associated to the use of HSC GT in neurodegenerative diseases.
See this image and copyright information in PMC

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