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)
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)
