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)
Stem Cell Researchers Demonstrate Safety Of Gene Therapy Using Adult Stem Cells May 8, 2008
Nolta and Bauer have worked on eighteen cell and gene therapy clinical therapy trials during their 12 years as colleagues, including stem cell gene therapy trials for adenosine deaminase deficiency, also known as the "Bubble Boy Disease," and stem cell gene therapy for HIV, with colleagues from Children's Hospital Los Angeles, who are also co-authors on the current report. Bauer is the director of UC Davis' Good Manufacturing Practice (GMP) laboratory in Sacramento. (Science Daily)
A rocky start, but Harrison's off home Aug 4, 2007
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. The disorder is also known as the "bubble-boy" disease, after an American boy who lived for 12 years in a plastic, germ-free bubble. (Sydney Morning Herald -- Australia)
Child undergoing stem cell transplant today May 25, 2007
ADA is the rarest form of SCID and occurs when the enzyme adenosine deaminase is lacking in the body, according to the National Center for Biotechnology Information. Sydnee's age and gender make her contraction of this disease even more uncommon. (Pekin Times, IL)
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)
Development of gene therapy Dec 24, 2006
In spite of continued great difficulties with technical implementation, initial successes in somatic gene therapy are on the horizon, for example in treating adenosine deaminase deficiency and chronic granulomatous disease. There are also initial indicators that point to the efficacy of gene therapy in treating chronic lymphatic leukaemia and haemophilia B. But successes in clinical testing come with their share of setbacks, such as the currently slight margin of therapeutic effectiveness over... (EurekAlert! -- Business News)
Stem Cell Therapy Debate Lives On, but Research Continues to Find Effective Applications in Immune and Organ Function Dec 11, 2006
In this study, a team of Italian researchers found that the use of stem cells may effectively fight SCID caused by a deficiency of the ADA gene (adenosine deaminase), which is critical for the immune system to function properly. Previous research has shown that immune function has improved when patients were given an autologous hematopoietic stem cell transplant (HSC), from the body's own bone marrow, combined with the ADA gene. (PR Newswire)
Parents agonize as trip delayed Nov 8, 2006
Without the adenosine deaminase (ADA), he is unable to filter out toxins that would kill off his immune system and eventually attack his organs. The injections work for only about two to three years. (Toronto Star -- Life)
W. French Anderson convicted Jul 21, 2006
As director of the Gene Therapy Laboratories at the University of Southern California's Keck School of Medicine in Los Angeles and a professor of molecular biology, biochemistry, and pediatrics, Anderson was known to many as the "father of gene therapy." In September 1990, he headed the first human gene therapy clinical protocol, successfully inserting the adenosine deaminase gene via retrovirus into the T lymphocytes of a 4-year-old girl with severe combined immunodeficiency, or SCID.. Anderson... (The Scientist)
A touching experience Jun 3, 2006
Parker has a rare form of SCID, which became known as the boy-in-the-bubble disease after a famous case in the 1970s, in which he's missing the enzyme adenosine deaminase (ADA) that removes toxins from his body. Without it, the toxins accumulate in his blood and organs and would lead to severe infections and organ death. (Toronto Star -- GTA)
Saving the 'bubble boy' May 20, 2006
When Parker arrives in Milan, doctors there will remove stem cells from his bone marrow, correct the DNA in them so that they contain a working gene that produces the enzyme adenosine deaminase (ADA), then reinject them into his bloodstream. They hope the stem cells will produce ADA, which removes toxins from the body. (Toronto Star -- Ontario)
