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Article

Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA <sup>−</sup> Immunodeficient Patients

20 Oct 1995

Abstract excerpt

Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic disorder treated by gene therapy. Two different retroviral vectors were used to transfer ex vivo the human ADA minigene into bone marrow cells and peripheral blood lymphocytes from two patients undergoing exogenous enzyme replacement therapy. After 2 years of treatment, long-term survival of T and B lymphocytes,...

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Identifiers

PMID: 7570000
DOI: 10.1126/science.270.5235.470

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

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Research trail→

Click a neighbor to travelStep 1 · 12 closest

Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA <sup>−</sup> Immunodeficient PatientsPMID 7570000Read ↗

T Lymphocyte-Directed Gene Therapy for ADA <sup>−</sup> SCID: Initial Trial Results After 4 YearsSemantic proximity Read ↗

Gene therapy for adenosine deaminase severe combined immune deficiency—An unexpected journey of four decadesSemantic proximity Read ↗

Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patientsSemantic proximity Read ↗

Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiencySemantic proximity Read ↗

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiencySemantic proximity Read ↗

An in Vivo Model of Somatic Cell Gene Therapy for Human Severe Combined ImmunodeficiencySemantic proximity Read ↗

Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild PreconditioningSemantic proximity Read ↗

Twenty-Five Years of Gene Therapy for ADA-SCID: From <i>Bubble Babies</i> to an Approved DrugSemantic proximity Read ↗

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.Semantic proximity Read ↗

Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trialSemantic proximity Read ↗

Successful Peripheral T-Lymphocyte–Directed Gene Transfer for a Patient With Severe Combined Immune Deficiency Caused by Adenosine Deaminase DeficiencySemantic proximity Read ↗

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapySemantic proximity Read ↗

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