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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CONCLUSION: Renal diseases and some of the main related diseases corresponded to 12.97% of the expenses in the triennium 2013-2015, and RRT to more than 5% of the SUS expenses with medium and high complexity healthcare. Such high expenses determine great concerns on the future maintenance of treatment for stage-5 CKD in Brazil and countries in similar or worse conditions of development. PMID: 29927463 [PubMed - as supplied by publisher]
Source: Jornal Brasileiro de Nefrologia - Category: Urology & Nephrology Tags: J Bras Nefrol Source Type: research
CONCLUSION: There was a significant increase in the number of transplants, with evolution in graft survival, despite the worsening in the profiles of recipients and donors. PMID: 29927459 [PubMed - as supplied by publisher]
Source: Jornal Brasileiro de Nefrologia - Category: Urology & Nephrology Tags: J Bras Nefrol Source Type: research
CONCLUSION: This analysis confirmed that in this population, infection is the leading cause of mortality over the first 5 years after kidney transplantation. Several demographic and socioeconomic risk factors were associated with death, most of which are not readily modifiable. PMID: 29927458 [PubMed - as supplied by publisher]
Source: Jornal Brasileiro de Nefrologia - Category: Urology & Nephrology Tags: J Bras Nefrol Source Type: research
Authors: Thanakitcharu P, Jitsuparat Y, Jirajan B Abstract Background: Patients with chronic kidney disease (CKD), end-stage renal disease (ESRD) with dialysis, and kidney transplant (KT) recipients are associated with increased cardiovascular (CV) morbidity and mortality. Arterial stiffness is a major nontraditional risk factor of CV disease, and increased aortic pulse wave velocity (PWV), the gold standard for arterial stiffness assessment, may predict CV morbidity and mortality in these patients. Objective: The purpose of the present study was to evaluate the prevalence of arterial stiffness in pre-dialysis ...
Source: Journal of the Medical Association of Thailand = Chotmaihet thangphaet - Category: General Medicine Tags: J Med Assoc Thai Source Type: research
Source: NanoFocus - Category: Nanotechnology Authors: 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
Written By Myra ChristopherMy mom was a steel magnolia (i.e., southern and perfectly charming), but she had a steel rod up her back. After her first surgery for stomach cancer at age 53, she refused pain medication because she said that she “could take it.” She was young and strong and committed to “beating cancer.” After nearly two years of chemotherapy, radiation and two more surgeries, the cancer won. Eventually, I watched her beg nurses to give her “a shot” minutes before another was scheduled and be told they were sorry but she would have to wait. I could tell by the expressions on ...
Source: blog.bioethics.net - Category: Medical Ethics Authors: Tags: Health Care chronic pain Opioid addiction Opioid Epidemic Opioid prescriptions syndicated Source Type: blogs
Scientists to begin trials for treatment to overcome inherited disease that affects 300,000 newborn babies a yearScientists are finalising plans to use gene therapy to treat one of the world ’s most widespread inherited diseases – sickle cell anaemia. The technique could begin trials next year, say researchers.About 300,000 babies are born globally with sickle cell disease. The condition causes red blood cells to deform, triggering anaemia, pain, organ failure, tissue damage, strokes and heart attacks. In the west, patients now live to their 40s thanks to the availability of blood transfusions and other treatme...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Sickle cell disease Society Health Genetics Biology Science Source Type: news
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