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Article

[Gene therapy for adenosine deaminase deficiency].

[Hokkaido igaku zasshi] The Hokkaido journal of medical science - 1 Jan 1996

Sakiyama Y

Abstract excerpt

Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency is a fatal recessive disorder caused by mutations in the gene encoding ADA. Based on the first clinical trial of two young girls with ADA-deficient SCID by recombinant retrovirus-mediated gene transfer at the National Institute of Health of USA, we prepared to treat a four-year-old boy with ADA-deficient SCID who had been treated...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 8727372

Genes

ADA

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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[Gene therapy for adenosine deaminase deficiency].PMID 8727372Read ↗

[Gene therapy for AIDS: current trends].Semantic proximity Read ↗

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 ↗

Treatment of Severe Combined Immunodeficiency Disease (SCID) due to Adenosine Deaminase Deficiency with CD34 <sup>+</sup> Selected Autologous Peripheral Blood Cells Transduced with a Human ADA Gene (Amendment). National Institutes of HealthSemantic proximity Read ↗

Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal productsSemantic proximity Read ↗

ADA-SCID Gene Therapy Endorsed By European Medicines Agency For Marketing AuthorizationSemantic 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 ↗

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

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

Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapySemantic proximity Read ↗

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

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