Navigation Links
'Bubble Boy' Kids Living Normally After Gene Therapy: Study
Date:8/24/2011

By Amanda Gardner
HealthDay Reporter

WEDNESDAY, Aug. 24 (HealthDay News) -- More than a dozen children with so-called "bubble boy" disease are alive and well, with functioning immune systems, nine years after undergoing gene therapy to correct their disorder, researchers report.

Most of the patients attend school with other children, something that probably would have been fatal without treatment.

"The promise of gene therapy is being fulfilled, at least for these diseases, where a number of patients are walking around in good health because they had gene therapy," said Dr. Donald Kohn, professor of microbiology, immunology and molecular genetics and pediatrics at the University of California, Los Angeles.

The disorder -- severe combined immunodeficiency (SCID) -- compromises the immune system so severely that children can't fight off normally innocuous infections. The condition is rare, and the term "bubble boy" was coined after a Texas boy with the condition lived in a germ-free plastic bubble.

Only boys inherit the gene in question, and many born with SCID die in infancy.

Two studies published Aug. 24 in Science Translational Medicine detail the results of the gene-modifying treatment. Kohn wrote a perspective piece accompanying the studies.

Traditionally, the only treatment for SCID was stem cell transplantation in which immune cells from a matching donor are transferred to the patient. But it's difficult to find matching donors and, even then, the patient's body may reject the transplanted cells.

With gene therapy, clinicians remove the patient's own bone marrow, isolate the stem cells, correct the gene and reinsert it into the patient, explained William J. Bowers, associate professor of neurology at the University of Rochester Medical Center in Rochester, N.Y.

The two current papers detail the success of gene therapy in two groups of patients: 10 boys with X-l
'/>"/>

Copyright©2010 ScoutNews,LLC.
All rights reserved  

Page: 1 2 3

Related medicine news :

1. Microbubbles to light the way to sentinel lymph nodes of breast cancer patients
2. Microbubble ultrasound and breast biopsies
3. Bursting bubbles with sound offers new treatments for cancer
4. Baby Bubbler a breath of fresh air
5. Gene Therapy Might Treat Bubble Boy Disease
6. The Acorn Aqua's Lead Free Bubbler
7. The proofs in the bubbles
8. New TB vaccine enters proof-of-concept trial in people living with HIV
9. Minimal scar techniques in living donors for kidney transplant
10. Genes, Not Healthy Living, Get Most to Age 100
11. Americans No Longer Outliving Europeans: Study
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
 'Bubble Boy' Kids Living Normally After Gene Therapy: Study
(Date:10/25/2014)... The Wehrman Collaborative, LLC (WeCo) ... disabled users by disabled users, has granted use ... Educational Resources of Burnsville, Minnesota, for their ... the Minnesota Alliance for Patient Safety of Minneapolis, ... website. Both organizations have met WeCo’s Standards ...
(Date:10/25/2014)... Raleigh, NC (PRWEB) October 25, 2014 ... patient who continued to receive pemetrexed (Alimta), after his ... to read the full story , just posted on ... study from Uji Tokushukai Medical Center in Japan suggests ... for mesothelioma patients who cannot tolerate higher ...
(Date:10/25/2014)... FRIDAY, Oct. 24, 2014 (HealthDay News) -- Researchers ... say their findings are cause for concern. ... that are raised for human consumption contribute to ... health, according to background information from the study. ... germs sicken about 2 million people and kill ...
(Date:10/22/2014)... 2014 At a time when the ... and athletes at all levels, the UPMC Sports ... current discussion where two powerful messages are lost: Concussions ... result in full recoveries every day. , In striving ... fact and research, UPMC and the Concussion Program are ...
(Date:10/22/2014)... October 22, 2014 For Dr. Iris Hunter, ... money; it’s an extension of her life’s mission. Her extensive ... as the owner of FirstLight HomeCare, make it clear that ... to me that my work makes a difference in the ... entrepreneurial spirit, so after much research, I felt ready to ...
Breaking Medicine News(10 mins):Health News:IT-Accessibility Award Granted to Education and Health Care for Section 508/WCAG 2Health News:IT-Accessibility Award Granted to Education and Health Care for Section 508/WCAG 3Health News:New Report Illustrates Extended Mesothelioma Survival with “Maintenance Therapy”, According to Surviving Mesothelioma 2Health News:Researchers Say Antibiotics in Fish a Health Concern 2Health News:University of Pittsburgh Medical Center Launches Initiative to Emphasize Concussions Are Treatable 2Health News:University of Pittsburgh Medical Center Launches Initiative to Emphasize Concussions Are Treatable 3Health News:University of Pittsburgh Medical Center Launches Initiative to Emphasize Concussions Are Treatable 4Health News:Dr. Iris Hunter to Host Grand Opening of FirstLight HomeCare Tomorrow 2
... Medical,Inc. (Nasdaq: NXTM ), a leading manufacturer of ... a $50.0 million credit,and security agreement from a group ... Merrill Lynch Business Services Inc., for a term of,42 ... million term loan and a $20.0,million revolving credit facility. ...
... Research Hospital have discovered a new signaling molecule that ... body. The finding could lead to the development of ... such as Type 1 diabetes; and for inflammatory diseases, ... , The St. Jude team discovered that specialized immune ...
... Conn., Nov. 21 Therap Services will host ... 5-7, 2007 in,Southbury, Connecticut. The 2nd Direct Support ... Support Professionals who are daily,users of Therap as ... Therap,s users and its developers and support staff., ...
... survey found , WEDNESDAY, Nov. 21 (HealthDay News) -- Older ... stress, a new study shows. , A team of University ... 53 to 85 who worked at least 20 hours per ... male, 87 percent were white, about 8 percent were black, ...
... Nov. 21 Susan G. Komen for the ... breast cancer research and,community-based outreach programs, is accepting ... who lost a,parent to breast cancer., (Logo: ... Award helps students cover the costs of,tuition, books, ...
... Region activates when a friend,s voice calls, scientists say , ... they,ve spotted a brain region that selectively emphasizes certain sounds, ... person,s name. , Researchers from the University of Washington, Seattle, ... the experimental method they used to detect brain activity associated ...
Cached Medicine News:Health News:NxStage Medical Establishes $50 Million Credit Facility with a Group of Lenders Led by Merrill Lynch Capital 2Health News:NxStage Medical Establishes $50 Million Credit Facility with a Group of Lenders Led by Merrill Lynch Capital 3Health News:St. Jude finds molecule that could improve cancer vaccines and therapy for other diseases 2Health News:St. Jude finds molecule that could improve cancer vaccines and therapy for other diseases 3Health News:Therap Services to Host 2nd Conference for Direct Support Professionals 2Health News:Jobs Don't Stress Most Older Workers 2Health News:Susan G. Komen for the Cure Provides Scholarships for Students Who Lost a Parent to Breast Cancer 2
(Date:10/25/2014)... -- Heska Corporation (Nasdaq: HSKA ) announces the ... Third Quarter 2014 Earnings Conference Call on the WebWhen:Wednesday, ... a.m. (EDT)Where: , www.heska.com (click on the ... of this website)How:Live over the Internet -- Simply log ... you may use the following teleconference access numbers:  Domestic: ...
(Date:10/25/2014)... Oct. 24, 2014   Metanome, Inc. , a ... services and data analysis, was the genomic sequencing provider ... Phase I/II clinical trial of SER 109, an Ecobiotic® ... Clostridium difficile infections (CDI). Metanome has ... R&D and clinical programs. Such genomic characterization has enabled ...
(Date:10/25/2014)... 2014 Best Practices designed the Medical ... a forum for sharing insights about the key ... mechanism for creating and sharing solutions through three ... from the Medical Affairs Consortium survey. ... the first roundtable of the 2014-2015 Medical Affairs ...
Breaking Medicine Technology:Webcast Alert: Heska Announces Third Quarter 2014 Earnings Conference Call Webcast 2Metanome Selected as Metagenomics Provider for Seres Health Clinical Trial 2Strong Participation in Best Practices' Medical Affairs Consortium Survey 2
... , KINGWOOD, Texas , ... (Pink Sheets: IMAI.PK) www.imaiinc.com announced today that ... purchase a minority interest in Assured Pharmacy, Inc. (Pink ... that is focused on selling prescription medications in the ...
... , WEST PALM BEACH, Fla. ... 50+ who have hearing loss, the AARP Hearing Care program, provided by HearUSA ... part of a nationwide rollout. , While one in 10 Americans have hearing ... suffer from this life altering condition. According to the National Institutes of ...
Cached Medicine Technology:International Merchant Advisors, Inc. Signs LOI to Invest in Pharmaceutical Chain 2International Merchant Advisors, Inc. Signs LOI to Invest in Pharmaceutical Chain 3HearUSA Introduces AARP Hearing Care Program for Americans Aged 50-plus in Ohio Today 2HearUSA Introduces AARP Hearing Care Program for Americans Aged 50-plus in Ohio Today 3HearUSA Introduces AARP Hearing Care Program for Americans Aged 50-plus in Ohio Today 4HearUSA Introduces AARP Hearing Care Program for Americans Aged 50-plus in Ohio Today 5
... 8F Percutaneous Vascular Surgical (PVS) System consists of ... Perclose Knot Pusher. The Prostar XL 8F PVS ... two pairs of sutured needles as well as ... precisely control the needles during deployment. the Perclose ...
... The new solid-state IRIS Medical IQ ... the treatment of retinal disorders and ... represents its latest technological innovation and ... Minimum Intensity Photocoagulation (MIP) procedures in ...
... The AIA-360 is a sophisticated Aumated Immunoassay Analyzer ... analyzer has a footprint of 16 X 16 ... Its compact design is ideal for physician ... testing, such as cardiac panels, as well as ...
... Solitaire represents the latest in green laser ... for use in both the operating room ... preference testing and the most experienced ophthalmic ... is the premium 532nm photocoagulator - unique ...
Medicine Products: