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Pioneering stem cell gene therapy cures infants with bubble baby disease

FINDINGSUCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.In a phase 2 clinical trial led by Dr. Donald Kohn of the  Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohn’s treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the child’s own c ells, has cured 30 out of 30 babies during the course of several clinical trials.BACKGROUNDAdenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzyme — a lifelong process that is very expensive and oft...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news

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Spark's first product, Luxturna, was approved by the FDA in December to treat an inherited retinal disorder that leads to blindness if untreated. The first patients have been treated with the gene therapy, which is now available at a select group of medical centers.
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
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Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
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Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
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Source: Medical Science Monitor - Category: Research Tags: Med Sci Monit Source Type: research
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Source: Stayin' Alive - Category: American Health Source Type: blogs
Two leading children ’s hospitals —UCLA Mattel Children ’s Hospital, part of UCLA Health, and  Miller Children ’s&Women ’s Hospital Long Beach, part of  MemorialCare Health System— announced today their intent to form a strategic affiliation that brings together their academic, clinical and research expertise, and resources to enhance children’s health care services in Southern California.The two organizations, which share similar missions and values, plan to establish a wider geographic pediatric collaboration that strengthens and broadens their ability to offer the ...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news
Follow me on Twitter @drClaire Every year, the Centers for Disease Control and Prevention (CDC) and the American Academy of Pediatrics (AAP) update the recommendations for immunizing children from birth to 18 years. This past week, the latest changes were published. The changes are usually small, and this year is no exception. But they are important — and they are a sign of how these organizations, and all the scientists who study immunization, take immunization effectiveness and safety very seriously. There is ongoing research to be sure that vaccines do everything we want them to do. As that research is done, disco...
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Source: blog.bioethics.net - Category: Medical Ethics Authors: Tags: Health Care chronic pain Opioid addiction Opioid Epidemic Opioid prescriptions syndicated Source Type: blogs
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