ADA-SCID


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Related to ADA-SCID: Adenosine deaminase deficiency
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Noun1.ADA-SCID - SCID resulting from mutation of a gene that codes for adenosine deaminaseADA-SCID - SCID resulting from mutation of a gene that codes for adenosine deaminase
SCID, severe combined immunodeficiency, severe combined immunodeficiency disease - a congenital disease affecting T cells that can result from a mutation in any one of several different genes; children with it are susceptible to infectious disease; if untreated it is lethal within the first year or two of life
References in periodicals archive ?
Revcovi is a new enzyme replacement therapy for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Babies with ADA-SCID need to be isolated in germ-free environments to prevent exposure to viruses and bacteria.
In 2016 the company was named a Fierce 15 Company by Fierce Biotech and was awarded a $19 million grant from the California Institute of Regenerative Medicine (CIRM) to advance their autologous ex vivo lentiviral gene therapy in ADA-SCID. In 2017, Orchard raised $110 million in a Series B round of funding to further develop its pipeline in parallel with enhancing manufacturing capabilities.
Also, Orchard and UCLA were awarded a $19 million grant from the California Institute of Regenerative Medicine (CIRM) in 2016 to advance the development of autologous ex-vivo lentiviral gene therapy in ADA-SCID.
In ADA-SCID, a single genetic defect prevents children from developing a vigorous immune system, leaving them unable to fight off ordinary infections.
Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement.
Interestingly, the plasma concentrations of adenosine in children with adenosine deaminase deficiency-severe combined immunodeficiency (ADA-SCID) are similar (6 [micro]mol/l) (6).
The boy, named only as Mustaf, suffered from the rare life-threatening immune disorder ada-SCID.
Mustaf was born in September 2000, and ada-SCID was diagnosed the following month.
It recommended less stringent criteria to allow resumption of the single gene therapy study aimed at a related immune system disorder called ADA-SCID, and also that the agency allow 27 distantly related gene therapy experiments to proceed under strict informed consent guidelines and closely followed for signs of leukemia.
In the field of Rare Diseases, Strimvelis became the first ex-vivo stem cell gene therapy to be approved for patients with the very rare immunodeficiency disease ADA-SCID.