HOME >> BIOLOGY >> NEWS
Switching off Huntington's

USING gene therapy to switch off genes instead of adding new ones could slow down or prevent the fatal brain disorder Huntington's disease. The method, which exploits a mechanism called RNA interference, might also help treat a wide range of other inherited diseases.

"When I first heard of this work, it just took my breath away," says Nancy Wexler of Columbia University Medical School, who is president of the Hereditary Disease Foundation in New York. Though the gene-silencing technique has yet to be tried in people, she says it is the most promising potential treatment so far for Huntington's.

It involves a natural defence mechanism against viruses, in which short pieces of double-stranded RNA (short interfering RNAs, or siRNAs) trigger the degradation of any other RNA in the cell with a matching sequence. If an siRNA is chosen to match the RNA copied from a particular gene, it will stop production of the protein the gene codes for (see Graphic).

Huntington's is caused by mutations in the huntingtin gene. The resulting defective protein forms large clumps that gradually kill off part of the brain. Studies in mice have shown that reducing production of the defective protein can slow down the disease, and Beverly Davidson at the University of Iowa thinks the same could be true in people.

"If you reduce levels of the toxic protein even modestly, we believe you'll have a significant impact," she says. Late last year, her team showed that it is possible to reduce the amount of a similar protein by up to 90 per cent, by adding DNA that codes for an siRNA to rodent cells engineered to produce the protein.

The team was the first to use gene therapy to deliver such a payload, and they have now done the same with the huntingtin protein itself. Completely silencing the gene in people with the disease is not an option because brain cells may not survive without the protein. But we have two copies of most genes, and usually only one is defective in peop
'"/>

Contact: Claire Bowles
claire.bowles@rbi.co.uk
44-207-331-2751
New Scientist
12-Mar-2003


Page: 1 2

Related biology news :

1. Switching on the fly genome
2. World Congress on Huntingtons Disease to meet in Toronto, August 16-19, 2003
3. Link between neuronal calcium channel, mutated gene that causes Huntingtons disease identified
4. Fasting forestalls Huntingtons disease in mice
5. Novel gene mutation causes Huntingtons-like symptoms, providing window into how brain cells die
6. Bile acid inhibits cell death in Huntingtons disease
7. Stanford researchers home in on Huntingtons disease treatment
8. Scientists find solution to a mystery surrounding Alzheimers, Huntingtons and other neurological diseases
9. Using a mouse model, reserchers now understand changes occurring in the brain before the onset of Huntingtons disease
10. Hopkins researchers identify neurologic problem associated with motor disorders in Huntingtons disease
11. Weizmann researchers find evidence that links molecular mechanism to Huntingtons Disease

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:4/19/2016)... DUBAI , UAE, April 20, 2016 ... can be implemented as a compact web-based "all-in-one" system ... in the biometric fingerprint reader or the door interface ... requirements of modern access control systems. The minimal dimensions ... the ID readers into the building installations offer considerable ...
(Date:4/14/2016)... , April 14, 2016 ... and Malware Detection, today announced the appointment of ... the new role. Goldwerger,s leadership appointment comes ... the heels of the deployment of its platform at ... behavioral biometric technology, which discerns unique cognitive and physiological ...
(Date:3/31/2016)... , March 31, 2016  Genomics firm Nabsys has ... CEO, Barrett Bready , M.D., who returned to ... the original technical leadership team, including Chief Technology Officer, ... Product Development, Steve Nurnberg and Vice President of Software ... the company. Dr. Bready served as CEO ...
Breaking Biology News(10 mins):
(Date:6/27/2016)... ... June 27, 2016 , ... Newly created 4Sight Medical ... to the healthcare market. The company's primary focus is on new product introductions, ... strategies that are necessary to help companies efficiently bring their products to market. ...
(Date:6/24/2016)... , June 24, 2016 Epic ... sensitively detects cancers susceptible to PARP inhibitors by ... tumor cells (CTCs). The new test has already ... therapeutics in multiple cancer types. Over ... DNA damage response pathways, including PARP, ATM, ATR, ...
(Date:6/23/2016)... 2016   Boston Biomedical , an industry ... to target cancer stemness pathways, announced that its ... Drug Designation from the U.S. Food and Drug ... including gastroesophageal junction (GEJ) cancer. Napabucasin is an ... cancer stemness pathways by targeting STAT3, and is ...
(Date:6/23/2016)... Prostate Cancer Foundation (PCF) is pleased to announce 24 new Young ... cancer. Members of the Class of 2016 were selected from a pool of ... More About the Class of 2016 PCF Young Investigators ... ... ...
Breaking Biology Technology:
Cached News: