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Gene therapy could correct blood stem cells inside, rather than outside, the body

Year: 2025

Type: article

Abstract: Experiments in mice reveal an early postnatal window of opportunity for the effective transfer of genes to blood-cell-producing haematopoietic stem cells by injecting mice with gene-carrying lentiviral vectors. This approach showed therapeutic benefit in three mouse models of severe diseases, and could expand the applicability of haematopoietic stem-cell gene therapy in the clinic. An in vivo approach shows therapeutic benefit in three mouse models of severe disease.

Source: Nature

DOI: 10.1038/d41586-025-01779-5

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $12,690

OpenAlex ID: W4411210077

FWCI:

Cites: 5

Cited by:

Related to: 10