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1 month ago

News Source: cnn.com
Trial Underway In US Uses CRISPR Gene-editing In People With Severe Sickle Cell Disease
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Trial Underway In US Uses CRISPR Gene-editing In People With Severe Sickle Cell Disease

(CNN)Several dozen patients with sickle cell disease will be among the first in the United States treated for a genetic disease with the experimental gene-editing technology CRISPR.

Gene therapy helps patients avoid blood transfusion, study says The study aims to recruit up to 45 adults with severe sickle cell disease. Thompson said that many in the scientific community have been looking forward to the trial since it was posted in late 2018, though only Monday was a patient publicly identified in an interview with NPR. A representative for Vertex Pharmaceuticals, a co-sponsor of the CRISPR trial, declined CNN's request for an interview with an expert involved in the trial. The company did not disclose how far along the trial is but confirmed that it's still enrolling patients. Another co-sponsor, CRISPR Therapeutics, also declined to comment. People with sickle cell disease, a group of inherited blood disorders, have abnormal hemoglobin in red blood cells that can cause them to get "hard and sticky," clogging blood flow in small vessels, according to the US Centers for Disease Control and Prevention. Hemoglobin is a protein found in red blood cells that is responsible for carrying oxygen throughout the body. Approximately 100,000 Americans, most of African ancestry or identifying as black, are affected by the disease, which can result in pain, anemia, blindness, organ damage and a shorter average lifespan overall.

CRISPR gene-edited babies may be at increased risk of early death, study finds Thompson referred to other gene-editing technology called zinc-finger nucleases, which are also being investigated as a way to spur production of fetal hemoglobin.  A teen with sickle cell disease achieved complete remission using another method of gene editing, a lentivirus, at a children's hospital in Paris. Instead of focusing on fetal hemoglobin, this patient received an "antisickling" gene to replace the one that causes the disease. Thompson said eyes are on gene-editing therapies because many people "who have conditions like sickle cell disease could be cured by a bone marrow transplant, but most of us will not have a suitable matched donor to provide the bone marrow." Using gene editing, the patient becomes their own donor.

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