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).
... read moreLong-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.
... read moreCytoreductive 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.
... read moreNeurological, 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.
... read moreADA-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.
... read moreRenal 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.
... read moreTandem 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.
... read moreSpotlight 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.
... read moreCytoreductive 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.
... read moreSingle-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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