UCLA Stem Cell Researcher Pioneers Gene Therapy Cure for Children with “Bubble Baby” Disease
Posted: November 19, 2014 at 6:44 pm
Contact Information
Available for logged-in reporters only
Newswise UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called Bubble Baby disease, a life-threatening condition that if left untreated can be fatal within the first year of life.
The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patients own cells with no side effects.
To date, 18 children with SCID have been cured of the disease after receiving the stem cell gene therapy in clinical trials at UCLA and the National Institutes of Health.
All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in Life Sciences.
To protect children born with SCID they are kept in isolation, in controlled environments because without an immune system they are extremely vulnerable to illness and infection that could be lethal.
Other current options for treating ADA-deficient SCID are not always optimal or feasible for many children, said Kohn. We can now, for the first time, offer these children and their families a cure, and the chance to live a full healthy life.
Defeating ADA-Deficient SCID: A Game-Changing Approach
Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases, and in a child with ADA-deficient SCID even the common cold can prove fatal. The disease causes cells to not create an enzyme called ADA, which is critical for production of the healthy white blood cells that drive a normal, fully-functioning immune system. About 15 percent of all SCID patients are ADA-deficient.
Read more here:
UCLA Stem Cell Researcher Pioneers Gene Therapy Cure for Children with "Bubble Baby" Disease
- CAR T-Cell Immunotherapy for ALL - National Cancer Institute - October 27th, 2015
- Gene Therapy and Cell Therapy Defined | ASGCT - American ... - October 20th, 2015
- StemCell Therapy MD - October 3rd, 2015
- Stem Cell Therapy in Mexico - May 8th, 2015
- Autologous Adipose Tissue Derived Stromal Vascular Fraction Cells Application In Patients - Video - May 1st, 2015
- Platelet Rich Plasma Injections For Chronic Pain Relief May Help You Avoid Sugery - Video - May 1st, 2015
- Stem Cell Treatment for COPD | StemRx Bioscience Solutions - Video - April 25th, 2015
- Stem Cell Therapy Market in Asia-Pacific to 2018 - Video - April 22nd, 2015
- Phytoscience Philipine celebrity Share good effect of stem cell Therapy - Video - April 18th, 2015
- STEM CELL therapy incredible results for severe MS - Video - April 18th, 2015
- Clinical Benefits of Stem Cell Therapy - Video - April 18th, 2015
- NYC Health & Longevity Center Now Offering Stem Cell Therapy to Avoid Joint Replacement - April 13th, 2015
- U.S. Stem Cell Clinic: What Conditions Can Be Treated? - Video - April 12th, 2015
- Stem Cell Therapy In USA - April 12th, 2015
- Stem Cell Therapy for Pain - Now Available at Columbia Pain Management - Video - April 11th, 2015
- What is Stem Cell Therapy- Trinity Spine and Wellness Center New Port Richey - Video - April 11th, 2015
- U.S. Stem Cell Clinic: How long will my recovery take? - Video - April 11th, 2015
- U.S. Stem Cell Clinic: Stem Cell Banking - Video - April 11th, 2015
- U.S. Stem Cell Clinic: What is Stem Cell Therapy? - Video - April 11th, 2015
- U.S. Stem Cell Clinic: How is Stem Cell Therapy Performed? - Video - April 11th, 2015
- U.S. Stem Cell Clinic: When should I expect to see results? - Video - April 11th, 2015
- Can PRP and Stem Cell Therapy Help You? | Orlando Orthopaedic Center - Video - April 3rd, 2015
- Induced Pluripotent Stem Cell (iPSC) Industry Complete Report 2015 - 2016 - April 3rd, 2015
- Stem Cell Therapy for Neuromuscular Diseases | InTechOpen - March 31st, 2015
- Arizona Pain Stem Cell Institute Now Offering Stem Cell Therapy to Help Patients Avoid Hip and Knee Replacement - March 31st, 2015
- Center of Regenerative Orthopedics in South Florida Now Offering Stem Cell Therapy to Help Avoid Hip and Knee ... - March 31st, 2015
- Ask Dr. Lemper | Stem Cell Therapy Treatments - Video - March 29th, 2015
- No stem cell treatment for public servant's dodgy knee - March 26th, 2015
- Stem Cell Therapy - Lilli Donovan - Video - March 25th, 2015
- Stem Cell Research - Stem Cell Treatments - Treatments ... - March 25th, 2015
- What is "Stem cell therapy" ? | Steps in PRP Treatment : TV5 News - Video - March 23rd, 2015
- Stem cell clinics under govt scanner - March 22nd, 2015
- SuperCells at Centre for Life - Video - March 21st, 2015
- Knee arthritis 3 years after stem cell therapy by Harry Adelson, N.D. - Video - March 21st, 2015
- Stem Cell Injection Treatment Stem Cell Therapy - Video - March 21st, 2015
- stem cell therapy di indonesia Jakarta tangerang serpong bsd bintaro - Video - March 21st, 2015
- The Alpha Clinic for Cell Therapy and Innovation | City of Hope - Video - March 20th, 2015
- Amniotic Stem Cell Therapy Discussed by R3 (844) GET-STEM - Video - March 20th, 2015
- Stem cell therapy may help treat type 2 diabetes - March 20th, 2015
- CAR T-Cell Therapy - Nebraska Medicine - Video - March 18th, 2015
- Stem Cell Therapy- Sci Video2 - Video - March 18th, 2015
- Stem Cell Therapy Now Being Offered for NonHealing Wounds at Telehealth's Three Regenerative Medicine Clinics - March 17th, 2015
- Stem Cell Research & Therapy | Full text | Amnion-derived ... - March 15th, 2015
- High-Dose Immunosuppressive Therapy and Autologous ... - March 15th, 2015
- Miracle stem cell therapy reverses multiple sclerosis - Video - March 14th, 2015
- Hip and shoulder arthritis six months after bone marrow stem cell therapy by Harry Adelson ND - Video - March 13th, 2015
- Stem Cell Therapy Network - Video - March 12th, 2015
- Stem Cell Therapy Cream - Video - March 9th, 2015
- BioLogic Stem Cell Therapy Cream - Video - March 8th, 2015
- World Over - 2015-02-26 - Vatican latest, ISIS, stem cell therapy, Ray Flynn with Raymond Arroyo - Video - March 8th, 2015
- Shoulder Labral Tear Repair Using Stem Cell Therapy - Orthopedic Surgeon, Dr. Wade McKenna - Video - March 6th, 2015
- What Makes Riordan-McKenna Institute Different? Stem Cell Therapy for Orthopedics - Video - March 6th, 2015
- Overcoming Knee Replacement Surgery Faster Using Amniotic Tissue-Enhanced Stem Cell Therapy - Video - March 6th, 2015
- Stem Cell Therapy in Osteo Arthritis Knee - Video - March 6th, 2015
- stem cell therapy helps avoiding knee surgery - Video - March 6th, 2015
- Stem cell therapy in AKI Dr Mohamed Kamal - Video - March 6th, 2015
- Stem Cell Therapy for Bone Fractures - Board-Certified Orhopedic Surgeon, Wade McKenna DO - Video - March 5th, 2015
- Procedure Overview - The STEM CELL ORTHOPEDIC INSTITUTE of Texas - Video - March 5th, 2015
- Glen Wysoki at The STEM CELL ORTHOPEDIC INSTITUTE of Texas - Video - March 5th, 2015
- Shoulder/Hip torn labrum 2 years and 5 months (respectively) after stem cell therapy by Adelson - Video - March 1st, 2015
- Animal cells for a younger you - March 1st, 2015
- 'Miracle' stem cell therapy reverses multiple sclerosis - March 1st, 2015
- Stem cell therapy a boon to Parkinson's patients - February 27th, 2015
- Stem Cell Research & Therapy | Full text | Aromatic ... - February 23rd, 2015
- Why Stem Cell Therapy? - Video - February 22nd, 2015
- What is Bone Marrow Aspirate Concentrate (BMAC) in Stem Cell Therapy? - Video - February 21st, 2015
- Dr Ellis hosts seminar on Stem Cell Therapy & Facial Rejuvenation - Video - February 17th, 2015
- Hip arthritis 3.5 years after stem cell therapy by Harry Adelson, N.D. - Video - February 16th, 2015
- How about Stem Cell Therapy for LBD, Nova Cells Institute stem cells - Video - February 14th, 2015
- Stem Cell Therapy for Erectile Dysfunction - Alvarado Hospital - Video - February 14th, 2015
- Cell Therapy may have just raised $1M, but will crowdfunding have a lasting place in biotech? - February 10th, 2015
- International Stem Cell completes cell bank manufacturing for Parkinson's disease clinical trial - February 4th, 2015
- Tampa stem cell clinic is long on promises, not evidence - February 1st, 2015
- Denver Regenerative Medicine - What is regenerative medicine? - Video - January 30th, 2015
- Cell Trials - January 26th, 2015
- "She's Happy" RMG's next Stem Cell Miracle - Video - January 21st, 2015
- Nutech Mediworld - Pioneering human embryonic stem cell therapy - Video - January 21st, 2015
- Stem Cell Therapy | Recent Strides Quell Stem Cell Debate - Video - January 20th, 2015
- Dr Sherif Stem cell therapy on OA - Video - January 18th, 2015
- Live100 Hospitals - Stem Cell Therapy - Video - January 16th, 2015