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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.
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An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health. The researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatments for their condition, known as severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. The findings were published today in the New England Journal of Medicine.
ADA-SCID, which is estimated to occur in approximately 1 in 200,000 to 1,000,000 newborns worldwide, is caused by mutations in the ADA gene that impair the activity of the adenosine deaminase enzyme needed for healthy immune system function. This impairment leaves children with the condition highly susceptible to severe infections. If untreated, the disease is fatal
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.
Experimental gene therapy successfully treats children born without an immune system
An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves them without an immune system.
Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is caused by mutations in the ADA gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system. For children with the condition, even day-to-day activities like going to school or playing with friends can lead to dangerous, life-threatening infections. If untreated, ADA-SCID can be fatal within the first two years of life.