Mountain View High School rallies to help 10-month old Olivia Werner, who has a rare defect in her immune system. Nov 25, 2008
Olivia recently was diagnosed with Severe Combined Immunodeficiency Syndrome, known as SCID. Because of a defect in her immune system, she wasn't able to fight off bacteria and infections. She needed a bone-marrow transplant, and her mother was a match. (Fredericksburg.com, VA)
UCLA develops safer, more effective TB vaccine for HIV-positive people Oct 24, 2008
The researchers also showed that the new vaccine is much safer than BCG in a severely immunocompromised animal host mice with Severe Combined Immunodeficiency (SCID mice) that completely lack an immune system. While the vaccine could be administered to anyone, it is specifically designed to be given to HIV-positive newborns and adults whose immune systems are still relatively intact and are therefore able to mount a good immune response to the vaccine, including persons on antiretroviral... (EurekAlert!)
At Magee, cord blood donations translate into hope Oct 15, 2008
The curly headed 7-year-old from Portersville in Butler County was born with severe combined immunodeficiency, an extremely rare disease that makes children vulnerable to germs the rest of us fight off without even knowing it ... It was only after the Mayo doctors found that his immune cells were extremely dysfunctional that Children's doctors gave the Marshalls their diagnosis -- severe combined immunodeficiency. (Pittsburgh Post-Gazette, PA)
Stem cells from patients with genetic diseases Sep 7, 2008
Most of these diseases were Mendelian (single-gene) disorders, including Duchenne (DMD) and Becker muscular dystrophy (BMD), Huntington disease, Gaucher disease (GD) type III and adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID). In addition, cell lines were generated from individuals with the chromosomal disorder Down syndrome (trisomy 21) and the complex (multifactorial) conditions Parkinson disease and juvenile diabetes mellitus. (PHG Foundation)
Inhibitors Of The Molecule PI3K Throw One Form Of Leukemia A CurveB-ALL Aug 26, 2008
10, 2008) Severe combined immunodeficiency (SCID), sometimes called 'Boy in the bubble syndrome', is a genetic disorder in which the patient lacks most types of immune cell. Almost 10 years ago, two. (Science Daily)
Protein key to control, growth of blood cells Aug 14, 2008
For example, aplastic anemia, severe combined immunodeficiency disorders and hemoglobin disorders involve deficiencies of specific immune cells in the blood. Using a drug to inhibit Lnk could potentially produce larger numbers of HSCs for a successful bone marrow transplant. (EurekAlert!)
Disease-specific stem cells to boost research Aug 12, 2008
The full list of diseases represented: adenosine deaminase deciency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. Cell, 2008. (Ars Technica)
Why Gene Therapy Caused Leukemia In Some 'Boy In The Bubble Syndrome' Patients Aug 11, 2008
10, 2008) Severe combined immunodeficiency (SCID), sometimes called 'Boy in the bubble syndrome', is a genetic disorder in which the patient lacks most types of immune cell ... (May 1, 2000) French researchers have developed a method of gene therapy to treat human severe combined immunodeficiency (SCID) X1, a life-threatening disease inherited on the X chromosome ... 28, 2005) Two new studies show that newborn screening for Severe Combined Immunodeficiency (SCID) -- a rare, treatable disorder of... (Science Daily)
SEAN M. HAFFEY / Union-Tribune The race against gene doping Jul 28, 2008
In 2002 in France, doctors used gene therapy to treat 12 boys with X-linked severe combined immunodeficiency, or bubble-boy disease. The technique effectively treated SCID, but at least three boys developed leukemia and one died. (San Diego Union-Tribune)
ASK THE EXPERTSHow does gene therapy work? May 14, 2008
Out-of-the-body gene therapy has already been used to treat severe combined immunodeficiency also referred to as SCID or boy-in-the-bubble syndrome where patients are unable to fight infection and die in childhood. In this type of gene therapy, scientists use retroviruses, of which HIV is an example. (Scientific American)
"Bubble Boy" Has Leukaemia After UK Gene Therapy Dec 20, 2007
Doctors at Great Ormond Street Hospital said on Tuesday the boy had been successfully treated for x-linked severe combined immunodeficiency (SCID-X1) often known as "baby in the bubble syndrome," but developed leukaemia two years later. Although gene therapy has been linked to leukaemia before, this is the first case within the London hospital's programme. (MEDLINEplus)
Boy in a bubble gets leukaemia after gene therapy Dec 20, 2007
The boy who is now three was born with rare X-linked severe combined immunodeficiency, (SCID-X1), a condition which only affects males. It is often called the "baby in the bubble syndrome", and most victims die within their first year without treatment. (News-Medical.net)
Gene therapyThe implications of a patient developing leukaemia Dec 19, 2007
It stands for X-linked Severe Combined Immunodeficiency, and is also known as "bubble boy" disease. Children born with the condition, which is caused by a single mutated gene, have no immune system. (BBC News -- Health)
Child gets leukaemia after gene therapy Dec 19, 2007
Children with X-linked severe combined immunodeficiency, a rare condition that affects only males and is sometimes referred to as "baby in the bubble syndrome", die within their first year without treatment. The unidentified child is the first in the UK, but the fifth in Europe, to get leukaemia as a result of the treatment. (Guardian Unlimited)
New stem cell method used to cure mice with sickle cell anemia Dec 7, 2007
The strategy should work to treat hemophilia, thalassemia, and severe combined immunodeficiency disease, the "bubble boy" disease, according to researchers, and might also apply to disorders linked to mutations in a single gene, such as muscular dystrophy and cystic fibrosis. Scientists hope to use a similar approach to create cardiac cells to treat heart attack patients or nerve cells that could cure spinal cord injuries. (Boston Globe)
A rocky start, but Harrison's off home Aug 4, 2007
Harrison was born with a rare form of the genetic disorder known as severe combined immunodeficiency (SCID), which affects one in 2 million people. It is so rare that Harrison is the first person to be placed on an Australian register for adenosine deaminase deficiency, although it is thought there may be up to three cases. (Sydney Morning Herald -- Australia)
Gene therapy patient dies, trial shut down Jul 28, 2007
The only disease ever to be cured with gene therapy is "bubble boy disease," an immune disorder formally called severe combined immunodeficiency, or SCID. But gene therapy attempts are restricted to SCID patients who have no alternative, after doctors in 2003 discovered a few babies saved by gene therapy went on to develop cancer -- again, linked to the virus used to deliver the genes. Reflecting how seriously regulators take this latest death, the National Institutes of Health's advisory... (CNN -- Health)
Rx: Home Care May 26, 2007
In 1978, her daughter Julia was born with severe combined immunodeficiency, which required her to be kept in a completely sterile hospital environment--like the "boy in the bubble." When Julia was 3 months old, she had a bone-marrow transplant that left her with a functioning immune system but unable to breathe on her own. Her hearing was impaired as well. (Time.com)
Gene therapy aimed at curing blind children May 3, 2007
The safety of the treatment will be under particular scrutiny, following a gene therapy trial four years ago in which two children being treated for a rare immune disorder called Scid (severe combined immunodeficiency) developed leukaemia. Trials of the latest gene therapy in dogs have proved the treatment can improve and preserve vision enough for nearly blind animals to negotiate mazes. (Hindu)
Gene therapy aimed at restoring sight for 30,000 people May 2, 2007
The safety of the treatment will be under particular scrutiny, following a gene therapy trial four years ago in which two children being treated for a rare immune disorder called Scid (severe combined immunodeficiency) developed leukaemia. Their cancers arose when a virus used to deliver healthy genes accidentally led to tumour-promoting genes. (Guardian Unlimited)
The Doctor Is in a Rut Mar 21, 2007
When the child, named Shira, became desperately ill just days after arriving in the U.S., hospital physicians diagnosed her with severe combined immunodeficiency disorder (SCID), an inherited disease common in Asia. The only hope, they said, was a bone marrow transplant, a treatment so harsh it would put Shira's life at risk. (BusinessWeek)
HIV experiment offers hope in cystic fibrosis battle Mar 19, 2007
Another disease which looks ready to benefit from gene therapy techniques is severe combined immunodeficiency, a condition in which babies are born without an immune system. Bobby Gaspar, of Great Ormond Street hospital in London, who will also present his latest work at the meeting in Warwick, has used a genetic approach to treating the disease. (Guardian Unlimited)
Fragile boy defies odds, finds match Feb 17, 2007
Perhaps one in 75,000 to 100,000 babies is born with severe combined immunodeficiency disease, an inherited condition that renders tiny bodies incapable of fighting off infection and usually kills those afflicted by their first or second birthday. Nine-month-old Evan is one of those youngsters, forced to live in a sterile hospital room in Calgary, isolated from his family, awaiting a cure. (Globe and Mail)
Scientists map horse's DNA Feb 8, 2007
And Arabian horses can inherit a form of severe combined immunodeficiency, more commonly called "bubble boy" disease. So far, researchers have found more than 80 genetically linked diseases common to both horses and humans. (Newsday -- Health)
Colleagues upset by Anderson sentence Feb 6, 2007
While there he inserted the adenosine deaminase gene via retrovirus into the T lymphocytes of a 4-year-old girl with severe combined immunodeficiency, or SCID. More recently, Anderson has focused on gene therapy delivery systems, including virus-based vectors to transfer genes and better vectors to insert genes into hematopoietic stem cells. To date, Anderson has published almost 400 and been profiled by a number of publications, including the New York Times and Scientific American. (The Scientist)
A new step for newborn screening Jan 30, 2007
But as the disease - officially called severe combined immunodeficiency, or SCID - is proving, setting up a comprehensive system to diagnose, treat and care for babies born with devastating genetic disorders is anything but easy. And with New York and other states adopting a wait-and-see stance, advocates say the pressure for success in Wisconsin is considerable. (Newsday -- Health)
'Bubble boy' illness targeted Jan 26, 2007
Children's Hospital of Wisconsin, in conjunction with the Wisconsin State Laboratory of Hygiene and the Jeffrey Modell Foundation, is launching a pilot program to test newborns for severe combined immunodeficiency, or SCID. They will use surplus blood specimens from samples drawn as part of the state's current screening program. "This is a first-phase feasibility study," said Ronald Laessig, director of population health studies at the State Laboratory of Hygiene, which means the pilot program... (Seacoast New Hampshire)
Cells Passed From Mother To Child May Be First Step In Developing New Treatments For Type 1 Diabetes Jan 24, 2007
Maternal microchimerism first was recognized in children with severe combined immunodeficiency in the 1970s. In 1999, a study by Nelson et al was first to show that maternal microchimerism persists into adulthood for persons with uncompromised immune systems (Journal of Clinical Investigation 104:41-47). (Science Daily)
Wisc. to test for bubble boy disease Jan 16, 2007
Between 40 and 100 U.S. babies are estimated to be diagnosed each year with SCID, or severe combined immunodeficiency disease. The best-known victim was David Vetter, Houstons famous bubble boy who lived in a germ-proof enclosure until his death at age 12 in 1984. (MSNBC -- Health)
The Boy in the Bubble Jan 9, 2007
When David was born in 1971, his parents and doctors already knew that he would probably suffer from Severe Combined Immunodeficiency (or SCID), a rare genetic disease inherited from his mother that had already killed his older brother. This was the era before AIDS, before people realised how murderous an innocuous phrase such as "immune deficiency" could be. (Sydney Morning Herald -- Entertainment)
