ADA-SCID

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Related to ADA-SCID: Adenosine deaminase deficiency

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Noun

ADA-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

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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.

FDA Approves Revcovi, a New Enzyme Replacement Therapy for the Treatment of 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.

Gene Therapy Trial Cures Real-Life Bubble Boy

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.

GSK SIGNS AGREEMENT WITH ORCHARD THERAPEUTICS

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.

ORCHARD COMPLETES $110M SERIES B FINANCING

Only about a dozen children are born with ADA-SCID each year in Europe.

Gene-therapy cure has money-back guarantee; the most expensive drugs in history, or medicine's biggest bargains? Gene therapy could be both

In ADA-SCID, a single genetic defect prevents children from developing a vigorous immune system, leaving them unable to fight off ordinary infections.

Possible genetic stem cell cure for "Bubble Boy Disease" approved in Europe

Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement.

Adenosin deaminasa en el sindrome de inmunodeficiencia combinada severa

Interestingly, the plasma concentrations of adenosine in children with adenosine deaminase deficiency-severe combined immunodeficiency (ADA-SCID) are similar (6 [micro]mol/l) (6).

Endotoxin stimulated interleukin-10 production is enhanced by adenosine. Possible key to septic shock associated immune deficiency?

The boy, named only as Mustaf, suffered from the rare life-threatening immune disorder ada-SCID.

Scientists in gene therapy landmark

Mustaf was born in September 2000, and ada-SCID was diagnosed the following month.

Breakthrough brings fresh hope to 'bubble babies' in Britain

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.