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

Related Links:

At age 21, Katie Stubblefield became one of 40 people in the world to receive a face transplant
Source: Health News: CBSNews.com - Category: Consumer Health News Source Type: news
Publication date: Available online 14 August 2018Source: Nitric OxideAuthor(s): Li Wang, Luis E.F. Almeida, Sayuri Kamimura, Jack H. van der Meulen, Kanneboyina Nagaraju, Martha Quezado, Paul Wakim, Zenaide M.N. QuezadoAbstractSickle cell disease (SCD) patients can have limited exercise capacity and muscle dysfunction characterized by decreased force, atrophy, microvascular abnormalities, fiber distribution changes, and skeletal muscle energetics abnormalities. Growing evidence suggests that in SCD, there is alteration in nitric oxide (NO) availability/signaling and that nitrate/nitrite can serve as a NO reservoir and enha...
Source: Nitric Oxide - Category: Chemistry Source Type: research
Blood Purif 2018;46:323 –325
Source: Blood Purification - Category: Hematology Source Type: research
Authors: Singh N, Tripathi AK, Sahu DK, Mishra A, Linan M, Argente B, Varkey J, Parida N, Chowdhry R, Shyam H, Alam N, Dixit S, Shankar P, Mishra A, Agarwal A, Yoo C, Bhatt MLB, Kant R Abstract Previously, it has been stated that the BCR-ABL fusion-protein is sufficient to induce Chronic Myeloid Leukemia (CML), but additional genomic-changes are required for disease progression. Hence, we profiled control and tyrosine kinase inhibitors (TKI) alone or in combination with other drug-treated CML-samples in different phases, categorized as drug-sensitive and drug-resistant on the basis of BCR-ABL transcripts, the marke...
Source: Oncotarget - Category: Cancer & Oncology Tags: Oncotarget Source Type: research
Authors: Cajigas-Du Ross CK, Martinez SR, Woods-Burnham L, Durán AM, Roy S, Basu A, Ramirez JA, Ortiz-Hernández GL, Ríos-Colón L, Chirshev E, Sanchez-Hernandez ES, Soto U, Greco C, Boucheix C, Chen X, Unternaehrer J, Wang C, Casiano CA Abstract Patients with metastatic castration-resistant prostate cancer (mCRPC) develop resistance to conventional therapies including docetaxel (DTX). Identifying molecular pathways underlying DTX resistance is critical for developing novel combinatorial therapies to prevent or reverse this resistance. To identify transcriptomic signatures associated with ...
Source: Oncotarget - Category: Cancer & Oncology Tags: Oncotarget Source Type: research
Authors: Ignacio RAB, Lin LL, Rajdev L, Chiao E Abstract This review highlights current interventional clinical trials for HIV-associated malignancies (HIVAMs), with emphasis on 4 mechanistic areas: immunomodulatory therapies and gene therapies, including immune checkpoint inhibitors; cytotoxic therapies; novel tumor-targeted and virally targeted therapies in both AIDS-defining and non-AIDS-defining cancers (NADC); and other screening or topical/ablative interventions. A search on ClinicalTrials.gov located 35 trials, including 12 immunomodulatory or gene therapy trials, 6 cytotoxic therapy trials, 10 trials of the...
Source: Journal of the National Comprehensive Cancer Network : JNCCN - Category: Cancer & Oncology Tags: J Natl Compr Canc Netw 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
More News: Allergy & Immunology | Babies | Babies Heart Conditions | Bone Marrow Transplant | Cardiology | Children | Clinical Trials | Environmental Health | Food and Drug Administration (FDA) | Gene Therapy | Genetics | Grants | Heart | Heart Transplant | Hospitals | Lung Transplant | Microbiology | National Institutes of Health (NIH) | Pediatrics | Science | Sickle Cell Anemia | Stem Cell Therapy | Stem Cells | Study | Transplant Surgery | Transplants