| 单词 | ADA-SCID |
| 例句 | The disease at issue is severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. Column: California's stem cell program found a disease cure, but it's being blocked by a biotech firm 2021-05-24T04:00:00Z They broke through in 2016, when the European Commission approved a gene therapy for treating ADA-SCID, a rare immune disorder that often kills children before their first birthday. Gene therapy is facing its biggest challenge yet 2019-12-03T05:00:00Z Some gene therapies, such as one recently approved by European regulators to treat a severe genetic immune disorder called ADA-SCID, use a version of the technique. Targeted stem-cell attack could make transplants safer 2019-11-28T05:00:00Z In 2016, a drug called Strimvelis was approved in Europe for the treatment of a condition called adenosine deaminase severe combined immunodeficiency, or ADA-SCID, a form of the disease that afflicted David Vetter. Stem-cell and genetic therapies make a healthy marriage 2019-05-14T04:00:00Z That approach is also used in another gene therapy for a related disease called ADA-SCID. Experimental gene therapy frees ‘bubble-boy’ babies from a life of isolation 2019-04-16T04:00:00Z Deep in the announcement, Orchard said it would “reduce investment in ADA-SCID.” Column: California's stem cell program found a disease cure, but it's being blocked by a biotech firm 2021-05-24T04:00:00Z These first two therapies are for exceptionally rare conditions - GSK estimates there are only 15 new cases of ADA-SCID in Europe each year - but both drugs are expected to pave the way for bigger products. Gene Therapy Is Now Available, but Who Will Pay for It? 2017-08-08T04:00:00Z The treatment, developed at the San Raffaele, cures ADA-SCID, an immune disease that is often fatal in the first year of life. Fixing fate 2016-07-28T04:00:00Z In patients with ADA-SCID, an inherited mutation in the ADA gene affects the production of the adenosine deaminase enzyme, which is essential for immune-system function. Stem-cell and genetic therapies make a healthy marriage 2019-05-14T04:00:00Z The treatment is for an illness called ADA-SCID which prevents babies from fighting off everyday infections. Gene therapy drug approval granted to GSK - BBC News 2016-05-27T04:00:00Z Here’s what that means: Until Orchard performs the next clinical trial, disease victims can’t access the ADA-SCID treatment to which Orchard holds exclusive rights. Column: California's stem cell program found a disease cure, but it's being blocked by a biotech firm 2021-05-24T04:00:00Z It is aimed at children with a disease known as ADA-SCID, a rare condition in which a single genetic defect prevents sufferers from developing a robust immune system, leaving them very susceptible to infections. Glaxo’s Potential Cure for “Bubble Boy Disease” One Step Closer 2016-04-01T04:00:00Z Its Strimvelis treatment for ADA-SCID is also being lined up for submission to U.S. regulators, although Andrews said this would not happen before the end of next year. Europe gives green light to first gene therapy for children 2016-04-01T04:00:00Z For the treatment of ADA-SCID, stem cells are harvested from the patient, and a virus is used to insert a healthy version of the ADA gene. Stem-cell and genetic therapies make a healthy marriage 2019-05-14T04:00:00Z Hot on Glybera’s heels is another gene therapy treatment which targets the immunodeficiency disorder ADA-SCID. Europe Nears First Approval for Gene Therapy Treatment 2012-07-23T17:15:04.197Z Indeed, in its latest quarterly financial disclosure, it listed the expense associated with restarting the ADA-SCID trial among the “risk factors” investors should consider. Column: California's stem cell program found a disease cure, but it's being blocked by a biotech firm 2021-05-24T04:00:00Z They also hope to test the new vectors in ADA-SCID. Gene-Therapy Successes Spur Hope for Embattled Field 2011-08-25T10:15:05.227Z |
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