Lou Portero

Urban Health Today

Today, a 12-year-old boy made medical history as the recipient of the first successful gene therapy for sickle cell disease (SCD). The breakthrough, according to a recent New York Times article, signals a promising new chapter for individuals grappling with this debilitating blood disorder.Researchers utilized CRISPR-Cas9 gene editing technology to correct the genetic mutation responsible for SCD. This involves modifying the patient&rsquo;s own hematopoietic stem cells outside the body to produce functional hemoglobin. These corrected cells are then reintroduced into the patient&rsquo;s bloodstream, where they can replenish the supply of healthy red blood cells.This new process is not without its drawbacks. The manufacturer estimates it can treat the cells of only 85 to 105 patients each year. In addition, the process costs $3.1 million for the gene therapy. The therapy, named Lyfgenia, is the most expensive treatment of its kind.Medical centers also have the capacity to handle only a limited number of gene therapy patients. Each person needs expert and intensive care. After a patient&rsquo;s stem cells have been treated, the patient has to stay in the hospital for a month. For most of that time, patients are severely ill from powerful chemotherapy.Children&rsquo;s National, the center in which this first therapy is being performed has capacity for only about 10 gene therapy patients a year.&ldquo;This is a big effort,&rdquo; said Dr. David Jacobsohn, chief of the medical center&rsquo;s division of blood and marrow transplantation.While still in its early stages, the success of CRISPR-based treatments for sickle cell disease opens the door to potential cures for other genetic disorders. Researchers are optimistic about the possibility of applying similar strategies to conditions such as beta-thalassemia, cystic fibrosis, and muscular dystrophy, offering hope to patients and their families worldwide.&ldquo;Sickle cell always steals my dreams and interrupts all the things I want to do,&rdquo; said 12-year-old Kendric Cromer, who is now hopeful about a chance for a normal life. &rdquo;I want to be cured. I want to play basketball.&rdquo;&nbsp;Source: <a href="https://www.nytimes.com/2024/05/06/health/sickle-cell-cure-first.html?campaign_id=60&amp;emc=edit_na_20240506&amp;instance_id=0&amp;nl=breaking-news&amp;ref=cta&amp;regi_id=90465757&amp;segment_id=165770&amp;user_id=213868c1fea37892c449528f649e5c4b">NY Times</a>

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Sickle Cell Disease

a 12-year-old boy made medical history as the recipient of the first successful gene therapy for sickle cell disease

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