Credit: Ann Johansson/UCLA Broad Stem Cell Research Center
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.
Massachusetts Institute of Technology
In early 2020, a few months after the Covid-19 pandemic began, scientists were able to sequence the full genome of SARS-CoV-2, the virus that causes the Covid-19 infection. While many of its genes were already known at that point, the full complement of protein-coding genes was unresolved.
Now, after performing an extensive comparative genomics study, MIT researchers have generated what they describe as the most accurate and complete gene annotation of the SARS-CoV-2 genome. In their study, which appears today in Nature Communications, they confirmed several protein-coding genes and found that a few others that had been suggested as genes do not code for any proteins.
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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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CAMBRIDGE, MA In early 2020, a few months after the Covid-19 pandemic began, scientists were able to sequence the full genome of the virus that causes the infection, SARS-CoV-2. While many of its genes were already known at that point, the full complement of protein-coding genes was unresolved.
Now, after performing an extensive comparative genomics study, MIT researchers have generated what they describe as the most accurate and complete gene annotation of the SARS-CoV-2 genome. In their study, which appears today in
Nature Communications, they confirmed several protein-coding genes and found that a few others that had been suggested as genes do not code for any proteins.
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