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: The described tunneling flap approach allows a hermetic soft tissue closure, characterized by a reduction of dehiscence and a secure bone graft healing. The combination of thin autogenous bone blocks and bone particles according to the SBB technique allows an acceleration of transplant revascularization, and thus, of graft regeneration, allowing a shortening of the patient treatment time as well as long-term three-dimensional volumetric bone stability. PMID: 30883623 [PubMed - in process]
Source: International Journal of Oral and Maxillofacial Implants - Category: ENT & OMF Tags: Int J Oral Maxillofac Implants Source Type: research
Authors: Sartori EM, das Neves AM, Magro-Filho O, Mendonça DBS, Krebsbach PH, Cooper LF, Mendonça G Abstract Regulation of cellular function is key to bone formation at endosseous implant surfaces. Osseointegration was "discovered" prior to the discovery of genetic regulation of osteoinduction or characterization of mesenchymal stem cells. Understanding osseointegration in cellular and molecular terms has benefited from genome-wide characterization of this healing process at endosseous implants in vivo. These in vivo studies also demonstrate a role for osteoprogenitor cells and cells involve...
Source: International Journal of Oral and Maxillofacial Implants - Category: ENT & OMF Tags: Int J Oral Maxillofac Implants Source Type: research
Abstract Over the past few decades, there was an encouraging breakthrough in bridging the gap between advancements in the evolution of diagnosis and treatment towards a better outcome in achalasia. The purpose of this review is to provide updated knowledge on how the current evidence has bridged the gap between advancements in the evolution of diagnosis and treatment of esophageal achalasia. The advent of high-resolution manometry and standardization based on the Chicago classification has increased early recognition of the disease. These 3 clinical subtypes of achalasia can predict the outcomes of patients, and t...
Source: Biomed Res - Category: Research Authors: Tags: Biomed Res Int Source Type: research
In this study, we investigated the efficacy of three parts (fruit, stem, and leaf) of Lonicera caerulea var. emphyllocalyx extract (LCEEs) against murine S. pyogenes infection. Oral administration of LCEEs increased the mortality in murine model, and the extracts of its stems and leaves were more effective than the fruit extract significantly. Murine splenocytes and mesenteric lymph nodal cells treated with LCEEs suppressed the excess production of inflammatory cytokine such as TNF-α in comparison to those from untreated cells. LCEEs stimulated the differentiation of pluripotent hematopoietic stem cells in those muri...
Source: Biomed Res - Category: Research Authors: Tags: Biomed Res Int Source Type: research
Source: Pediatric Nephrology - Category: Urology & Nephrology Source Type: research
A Chinese scientist shocked the scientific community a couple of days ago with the announcement of having modified the very blueprint of life. If his claims are true, he tried to bestow two baby girls the ability to resist possible future infections with HIV. The outrage shows that humanity is not prepared to utilize the power of gene editing on embryos yet. We have no idea about the biological consequences, and we haven’t tackled the necessary legal and ethical issues. Genes to become toys of the “Gods”? Humanity has come a long way since Aldous Huxley pinned down how methods of genetic engineering, bio...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Bioethics Future of Medicine Genomics designer babies designer baby Gene gene editing genes Genome genome sequencing Health Healthcare healthcare system Innovation technology Source Type: blogs
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
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