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An international team of researchers at Great Ormond Street Hospital (GOSH), and University of California, Los Angeles (UCLA) have developed a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency that leaves them without an immune system.
Severe combined immunodeficiency due to adenosine deaminase deficiency, also known as ADA-SCID, is a rare, life-threatening disease that prevents children from living a normal life. It is caused by mutations in the gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system.
Children with ADA-SCID have no immune system and, if left untreated, the condition can be fatal within the first two years of life. Day-to-day activities like going to school or playing with friends can lead to a dangerous infection. Recently, new-born screening for SCID has been implemented in some countries to help diagnose the condition early in life.
Study suggests gene therapy as promising cure
ANI
11 May 2021, 23:56 GMT+10
California [US], May 11 (ANI): Researchers have developed a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency that leaves them without an immune system.
The study led by an international team of researchers at Great Ormond Street Hospital (GOSH), and the University of California, Los Angeles (UCLA) - was published in the New England Journal of Medicine.
Severe combined immunodeficiency due to adenosine deaminase deficiency, also known as ADA-SCID, is a rare, life-threatening disease that prevents children from living a normal life. It is caused by mutations in the gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system.
Job Research Foundation Announces 2021 Grant Recipients; Funding will Support Research into the Causes of and Treatments for Job Syndrome
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Job Research Foundation has announced the recipients of its third round of grant funding to support investigation into the causes of and treatments for Job Syndrome, a rare multi-system immunodeficiency disorder. To date the organization has funded a total of ten research projects across the world.
Our vision is to help find a cure for Job Syndrome by providing the scientific community with additional opportunities to further research the disease. NEW YORK (PRWEB) February 22, 2021
Job Research Foundation has announced the recipients of its third round of grant funding to support investigation into the causes of and treatments for Job Syndrome. The Foundation has awarded two-year grants of $200,000 each to support two research projects. To date the organization ha
The University of Southampton is hosting a free virtual panel event to discuss the work Southampton is doing in the battle against COVID-19.
The event ‘Beating COVID-19: Trials, Vaccinations & Prevention’ will take place at 18:00 on February 9th. It will feature leading University faculty members discussing their roles in the COVID-19 fight and an opportunity for viewers to ask their burning question.
Professor John Holloway will host the event. The host is a professor of Allergy and Respiratory Genetics and also Associate Dean (Research) in the Faculty of Medicine at the University of Southampton.
New event announcement!
Beating COVID-19: Trials, Vaccinations and Prevention.@ProfJWHolloway welcomes Professors Saul Faust, @profrobread and Lucy Yardley who will deliver a panel discussion about @unisouthampton s invaluable involvement in the COVID-19 pandemic.