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Progress in Genetic Therapy for Severe Combined Immunodeficiency Associated With Adenosine Deaminase Deficiency

Year: 2009

Type: article

Abstract: A recent report in the New England Journal of Medicine provides details of the long-term follow-up of 10 patients treated with stem cell gene therapy for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.1 The results show significant success in reversal of the life-threatening immunodeficiency phenotype and a lack of the insertional mutagenesis that was reported in both the X-linked SCID (X-SCID)2,3 and chronic granulomatous disease (CGD)4 trials. Overall, the report shows outcomes in this disease that compare very favorably with current stem cell transplantation and enzyme-replacement therapeutic modalities.

Source: Molecular Therapy

DOI: 10.1038/mt.2009.57

Author: David A Williams

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: $4,500

OpenAlex ID: W1967023974

FWCI:

Cites: 6

Cited by: 1

Related to: 10