medwireNews: A report published in Human Gene Therapy Clinical Development describes the preclinical safety studies conducted to support the European market authorisation application for GSK2696273, a gammaretroviral-based gene therapy for adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID).
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Long-term enzyme replacement therapy feasible in ADA-deficient patients
medwireNews: Enzyme replacement therapy over the long term could be an option for patients with adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) for whom curative therapies are unavailable, say US researchers who describe a patient who has remained on treatment for 24 years.
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Cytoreductive conditioning intensity linked to post-gene therapy clonal, TCR diversity
medwireNews: In patients with adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID) receiving gammaretroviral gene therapy, the intensity of cytoreductive conditioning could have a bearing on the success of the procedure, in terms of clonal diversity and the T-cell receptor (TCR) repertoire, research indicates.
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Neurological, behavioural defects an intrinsic manifestation of ADA deficiency
medwireNews: Neurological and behavioural impairments are not uncommon in patients with adenosine deaminase deficient-severe combined immunodeficiency (ADA-SCID), report Italian researchers who propose aberrant adenosine metabolism in the brain as the underlying cause.
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ADA-SCID testing can be incorporated in routine neonatal screening
medwireNews: An Italian research team has developed an analytical method that enables the inclusion of adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) testing in a tandem mass spectrometry-based newborn screening programme.
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Renal manifestation of ADA deficiency identified
medwireNews: Adenosine deaminase (ADA) deficiency can present as atypical haemolytic uraemic syndrome (HUS), say researchers who describe four such cases in the Journal of Clinical Immunology.
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Tandem mass spectrometry detects delayed-onset ADA-SCID
medwireNews: Tandem mass spectrometry (MS), but not T-cell receptor excision circle (TREC) analysis, can identify newborns likely to develop delayed- or late-onset adenosine deaminase–severe combined immunodeficiency (ADA-SCID), findings indicate.
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Spotlight on haematopoietic stem cell transplantation in ADA-SCID patients
medwireNews: A multicentre review of patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) treated with haematopoietic stem cell transplantation (HCT) details the outcomes of these patients.
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Cytoreductive conditioning essential for maximising gene therapy benefits
medwireNews: The outcomes of two patients with adenosine deaminase (ADA)-deficiency who underwent stem cell gene therapy (SCGT) without cytoreductive conditioning reinforce the importance of cytoreduction to ensure maximal benefits from gene therapy, report investigators.
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Single-centre experience of ADA-deficient patients charted
medwireNews: A report published in the Journal of Clinical Immunology describes the genetic features and treatment course of children diagnosed with adenosine deaminase (ADA) deficiency at a single institution in Italy from 1997-2013. All 27 patients (14 female) were diagnosed at the Children’s Hospital in Brescia, with the majority displaying symptoms between 3 days to 7 months…
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