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.