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-linked SCID (SCID-X1); and six with ADA-SCID, which involves a slightly different gene mutation. All were between 6 months and 39 months old.

Gene therapy successfully treated four of the six ADA-SCID patients.

All the SCID-X1 children recovered, although one developed leukemia. That boy is currently in remission, but leukemia has been a problem with previous gene therapy trials.

Last year, French researchers reported that eight of nine male infants born with SCID-X1 had recovered as a result of gene therapy. Unfortunately, almost half developed acute leukemia, one of whom died.

The virus vector used in this earlier trial inadvertently activated an oncogene, which led to the development of the leukemia, researchers said.

The latest research circumvented this problem by using a different virus vector.

"A cloud was thrown over the field several years ago and they've solved it nicely," said Dr. Darwin Prockop, director of the Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine at Scott & White in Temple, Texas. "Very probably this can be used for other genetic diseases."

"This field came on with huge promise, then hit a few bumps and now . . . we're starting to see more and more of these successes," added Bowers.

More information

The U.S. National Human Genome Research Institute has more on SCID.

SOURCES: Donald B. Kohn, M.D., professor, microbiology, immunology and molecular genetics and pediatrics, University of California, Los Angeles; William J. Bowers, Ph.D., associate professor, neurology, University of Rochester Medical Center, Rochester, N.Y.; Darwin J. Prockop, M.D., Ph.D., director, Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine at Scott & White, Temple, Texas; Aug. 24, 2011, Science Translational Medicine


'/>"/>
Copyright©2010 ScoutNews,LLC.
All rights reserved  

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:1/13/2017)... ... 2017 , ... People who have sensitive teeth are about as common as ... teeth. Sadly, most dental hygiene products in the market contain chemical ingredients that only ... continuing their daily oral care routine to keep their teeth white and healthy is ...
(Date:1/13/2017)... , ... January 13, 2017 , ... Friday, January 13 ... of the game room, increasing the size of the location to 90,000 square feet. ... 8-hole mini golf course that takes customers on an educational tour of the historic ...
(Date:1/13/2017)... ... ... MyGenetx, a molecular laboratory headquartered in middle Tennessee is proud to announce ... LD, will serve on the 2017 TBCC Board. , Kris Wiese was ... year as Vice President. She will oversee the newly elected board of 17. Mary ...
(Date:1/13/2017)... CO (PRWEB) , ... January 13, 2017 , ... As ... a growing alliance of deans of colleges and schools of education across the country ... schools. , In a Declaration of Principles released today, 175 deans sounded the alarm: ...
(Date:1/13/2017)... ... ... Overseer at The House of Yahweh, has published a new article that refutes false accusations ... eternal joy. Yisrayl says he has been accused of being the “prophet of doom,” when ... are nothing new in his world and says it has always been that way for ...
Breaking Medicine News(10 mins):
(Date:1/12/2017)... India , January 12, 2017 ... titled, "IVD Quality Control Products Market by Product Type, by ... Opportunity Analysis and Industry Forecast, 2014 - 2022" The global ... in 2015, and is expected to reach $1,052 million by ... The quality controls products segment dominated the market with fourth-fifths ...
(Date:1/12/2017)... LONDON , Jan. 12, 2017 ... health benefits when consumed, which facilitate digestion, gastrointestinal ... There have been 500 million probiotics ... can realize 15% in some European countries. The ... in the developed countries. In developed countries, ...
(Date:1/12/2017)... , Jan. 12, 2017 "Global ... report analyzes ongoing clinical and non-clinical trends in ... are 3 lung cancer vaccines commercially available in ... trial of 30 lung cancer vaccines in clinical ... parameters associated with the development of the lung ...
Breaking Medicine Technology: