Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients.

doi:10.1016/j.ymthe.2025.05.034

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Article

Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Sept 2025

Hart Kevyn L, Crisostomo Ralph Valentine, Mittelhauser Annika, Zhan Lingyu, Kononov Nika, Bradford Kathryn, Kohn Donald B

Abstract excerpt

Adenosine deaminase severe combined immunodeficiency (ADA-SCID) is a monogenic disorder caused by mutations in the ADA gene. Gene therapy using γ-retroviral and lentiviral vector gene addition approaches have shown curative results. We sequenced the ADA transgene in transduced CD3+ T cells, and in peripheral blood cells from patients treated with autologous CD34+ cells transduced with either a γ-retroviral or...

Read the complete abstract on PubMed

Topics

Humans
Genetic Vectors
Lentivirus
Genetic Therapy
Mutation
HEK293 Cells

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Severe Combined Immunodeficiency

Adenosine Deaminase

Retroviridae

T-Lymphocytes

Transduction, Genetic

Transgenes

Agammaglobulinemia

APOBEC Deaminases

Source

Open in PubMed Open full text

Identifiers

PMID: 40450520
DOI: 10.1016/j.ymthe.2025.05.034
PMCID: PMC12432904

Genes

ADA
CD34

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 20 Oct 2026.

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