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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
As the nation’s opioid epidemic rages on, the federal agency charged with leading the government’s response to substance abuse is changing the way it helps local communities. But critics say the move risks leaving programs with fewer resources until the new plan is in place. The controversy began in January with a low-key announcement by the […]Related:E. coli outbreak spreads as source of tainted lettuce remains a mysteryFirst marijuana-derived drug poised for FDA approval after winning support from advisersHave you or someone you know waited too long for an organ transplant?
Source: Washington Post: To Your Health - Category: Consumer Health News Source Type: news
Mauro Di Ianni, Stefano Baldoni, Beatrice Del Papa, Patrizia Aureli, Erica Dorillo, Filomena De Falco, Elisa Albi, Emanuela Varasano, Ambra Di Tommaso, Raffaella Giancola, Patrizia Accorsi, Gianluca Rotta, Chiara Rompietti, Estev ão Carlos Silva Barcelos, Antonio Francesco Campese, Paolo Di Bartolomeo, Isabella Screpanti, Emanuela Rosati, Franca Falzetti, Paolo Sportoletti
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
Mayra A. Carrillo, Anjie Zhen, Scott G. Kitchen
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
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Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
CONCLUSIONS Our results suggest that gastric cancer cells induce the transition of pericytes to CAFs by exosomes-mediated BMP transfer and PI3K/AKT and MEK/ERK pathway activation, and suggest that pericytes may be an important source of CAFs. PMID: 29668670 [PubMed - in process]
Source: Medical Science Monitor - Category: Research Tags: Med Sci Monit Source Type: research
I suppose I should say something about the recently announced work ofShoukhrat Mitalipov of Oregon Health and Science University, who claims to have successfully edited the genome of human embryos, in this case to eliminate a disease causing mutation. This work is as yet unpublished and not peer reviewed, but let's assume it is sound.The technique, which has been much in the news, is calledCRISPR/Cas9.I'm not going to go into the technical details here but you can certainly look it up if you are interested,the Wikipedia article is actually reasonably accessible if you have some basic understanding of genetics. But getting ...
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...
Source: Harvard Health Blog - Category: Consumer Health News Authors: Tags: Children's Health Infectious diseases Parenting Prevention Vaccines Source Type: blogs
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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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