Navigation Links
Rare genetic disease successfully reversed using stem cell transplantation
Date:9/17/2009

sively worse with time. Other symptoms include diabetes, muscular disease, neurological dysfunction, and retinopathy. Infantile onset is the most common, as well as the most severe, form of the disease.

The only available drug to treat cystinosis, cysteamine, while slowing the progression of kidney degradation, does not prevent it, and end-stage kidney failure is inevitable.

"Cysteamine must be given every six hours, so children have to be woken up each night to take this drug, which has unpleasant side effects, and many others to treat various symptoms," Cherqui says. "So although there is treatment, it is difficult treatment that does not cure the disease."

"Surprised and Encouraged"

In the new study, the researchers found that transplanted bone marrow stem cells carrying the normal lysosomal cystine transporter gene abundantly engrafted into every tissue of the experimental mice. This led to an average drop in cystine levels of about 80 percent in every organ. In addition to preventing kidney dysfunction, there was less deposition of cystine crystals in the cornea, less bone demineralization, and an improvement in motor function.

"The results really surprised and encouraged us," says Cherqui, who as a doctoral student in France in 1998 helped discover the gene involved in cystinosis. "Because the defect is present in every cell of the body, we did not expect a bone marrow stem cell transplant to be so widespread and effective."

Cherqui, who generated the mouse model in 2000 that is currently used to study cystinosis, says that adult bone marrow stem cell therapy is particularly well suited as a potential treatment for cystinosis because these cells target all types of tissues. In addition, stem cells reside in the bone marrow for the duration of a patient's life, becoming active as needed, a particular benefit for a progressive disease like cystinosis.

The work of Cherqui and her colleague
'/>"/>

Contact: Keith McKeown
kmckeown@scripps.edu
858-784-8134
Scripps Research Institute
Source:Eurekalert

Page: 1 2 3

Related biology news :

1. Does the desire to consume alcohol and tobacco come from our genetic makeup?
2. Diverse genetic abnormalities lead to NF-κB activation in multiple myeloma
3. Many parents at-risk for cancer disclose genetic test results to children
4. Genetics determine optimal drug dose of common anticoagulant
5. Claims of sex-related differences in genetic association studies often not properly validated
6. American College of Medical Genetics responds to new FDA labeling decision for warfarin
7. UNC study questions FDA genetic-screening guidelines for cancer drug
8. Genome study shines light on genetic link to height
9. Selexis Announces Advanced Approach to Maximize Power of Genetic Elements for Rapid Development of High Performance Cell Lines
10. Genes, Environment and Health Initiative invests in genetic studies, environmental monitoring
11. Rutgers Genetics receives $7.8 million for autism research
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/23/2014)... news release is available in German . ... and stability to cells, drive metabolic processes and transmit ... complex three-dimensional shapes. Scientists at the Max Planck Institute ... proteins can be constructed of similar amino acid chains ... that the proteins that exist today arose from common ...
(Date:7/23/2014)... BioCatch, the global leader in ... has been named in Gartner,s Market Guide for Online ... well-known Magic Quadrant for Web Fraud Detection. ... several vendors to deliver passive biometric analysis and predicted, ... feature of at least 30% of one-stop fraud detection ...
(Date:7/23/2014)... clinical use of induced pluripotent stem cell (iPSC) ... hindered by the risk of dysregulated cell growth, ... use etoposide treatment to halt teratoma formation in ... acute myocardial infarction, is demonstrated in an article ... peer-reviewed journal from Mary Ann Liebert, Inc., publishers. ...
Breaking Biology News(10 mins):Protein evolution follows a modular principle 2BioCatch Featured in Gartner's 2014 Online Fraud Detection Market Guide 2BioCatch Featured in Gartner's 2014 Online Fraud Detection Market Guide 3
... January 2014 Scientists from the Montreal Neurological ... two genes linked to hereditary Parkinson,s disease are ... The protective mechanism, which is reported in T ... arise from oxidative stress from mitochondria., "PINK1 and ...
... 21, JoVE, the Journal of Visualized Experiments , ... explored one of the most intriguing research questions facing ... protocol, titled Conducting Miller-Urey Experiments, is comprised of a ... Dr. Stanley Miller and Dr. Harold Urey in 1953. ...
... viral pathogen that typically infects plants has been found ... Researchers working in the U.S. and Beijing, China report ... journal of the American Society for Microbiology. The ... "resulted in the serendipitous detection of Tobacco Ringspot Virus, ...
Cached Biology News:Quality control of mitochondria as a defense against disease 2A 21st century adaptation of the Miller-Urey origin of life experiments 2Pathogenic plant virus jumps to honeybees 2
(Date:7/23/2014)... 23, 2014 StemGenex® , the ... the US aimed at improving the lives of patients ... study for Parkinson’s disease. StemGenex believes that a ... therapy are paramount when providing care to patients with ... stem cell therapy accessible to the millions of individuals ...
(Date:7/23/2014)... 2014 /PRNewswire/ - BioAmber Inc. (NYSE: ... chemicals, today announced that the underwriters of its common ... purchase an additional 420,000 shares of common stock ... share, less underwriting discounts and commissions, in connection ... 2,800,000 shares of common stock, bringing the expected ...
(Date:7/23/2014)... , July 23, 2014  GrowBLOX Sciences, Inc. (OTCQB: ... that it is filing for two medical marijuana dispensary locations ... are in addition to the Clark County, Nevada ... special use permit, as previously announced.  GrowBLOX Sciences, ... be submitting two new applications for dispensaries in the ...
(Date:7/23/2014)... With the ability to record continuously at over ... 15 minutes, Fastec Imaging has brought the same ease-of-use of ... “Finally, a high speed camera that can be used as ... lives.” proclaims Matt Kearney, VP of Sales for Fastec Imaging. ... and simplify the traditionally complicated workflow of a high speed ...
Breaking Biology Technology:StemGenex® Gives Hope to Parkinson’s Patients through New Stem Cell Clinical Study 2StemGenex® Gives Hope to Parkinson’s Patients through New Stem Cell Clinical Study 3BioAmber announces exercise in full of underwriters' option in connection with previously priced follow-on offering 2BioAmber announces exercise in full of underwriters' option in connection with previously priced follow-on offering 3GrowBLOX Sciences, Inc. Files for Two Additional Medical Marijuana Dispensaries in Las Vegas 2GrowBLOX Sciences, Inc. Files for Two Additional Medical Marijuana Dispensaries in Las Vegas 3Fastec Imaging Introduces the First High Speed Camcorder 2Fastec Imaging Introduces the First High Speed Camcorder 3
... /PRNewswire-FirstCall/ - On August 20, 2008,Oncothyreon Inc. (Nasdaq: ... a,letter from The Nasdaq Stock Market indicating that (i) ... on The NASDAQ Global Market,because it did not meet ... other things, that the market value of,its common stock ...
... with international ... and technology companies, TORONTO, Sept. 17 Aurillion, a ... Moreno as its vice president of sales and marketing. In ... grow the company,s business throughout the Americas, Europe,and Asia. He ...
... 17 Sigma-Aldrich Corporation,(Nasdaq: SIAL ) will be ... Wednesday, September 24th at 8:30 AM ET at the ... via live audio broadcast over the,Internet available at ... this file. For the webcast on, http://ir.sigmaaldrich.com users ...
Cached Biology Technology:Oncothyreon announces receipt of NASDAQ letter regarding compliance with NASDAQ Global Market listing standards 2Oncothyreon announces receipt of NASDAQ letter regarding compliance with NASDAQ Global Market listing standards 3Guillermo Moreno Named Aurillion's Vice President of Sales and Marketing 2
... Roches RTS AviTag E. coli Biotinylation Kit, Linear ... 100 E. coli expression kits. (Cat. No. 3 ... reagents to rapidly generate linear expression constructs by ... based on a combination of T7 RNA polymerase ...
... (GR) Competitor Assay Kits are ideal for ... The PolarScreen GR Competitor Assay Kits contain ... and one of the following proprietary fluorescent ... GS Red (red assay kit), or GS ...
... E. coli Biotinylation Kit, Plasmid is designed for ... coli expression kits. (Cat. No. 3 186 148, ... provides vectors for the easy introduction of a ... protein can be enzymatically mono-biotinylated by E. coli ...
... Exiqons LNA technology enables sensitive, fast ... on intact chromosomes. Short hybridisation ... fixed cells and chromosome spreads Multiplex ... Pre-validated probes Probes available ...
Biology Products: