Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

UCLA study identifies 2 chemicals that could lead to new drugs for genetic disorders

30.09.2009
Discovery could help people with cancer, muscular dystrophy, A-T

UCLA scientists have identified two chemicals that convince cells to ignore premature signals to stop producing important proteins. Published in the Sept. 28 edition of the Journal of Experimental Medicine, the findings could lead to new medications for genetic diseases, such as cancer and muscular dystrophy, that are sparked by missing proteins.

"When DNA changes, such as nonsense mutations, occur in the middle rather than the end of a protein-producing signal, they act like a stop sign that tells the cell to prematurely interrupt protein synthesis," explained Dr. Richard Gatti, professor of pathology and laboratory medicine and human genetics at the David Geffen School of Medicine at UCLA. "These nonsense mutations cause the loss of vital proteins that can lead to deadly genetic disorders."

Gatti's lab specializes in studying ataxia-telangiectasia (A-T), a progressive neurological disease that strikes young children, often killing them by their late teens or early 20s.

For four years, the UCLA Molecular Shared Screening Resources Center of the campus' California NanoSystems Institute has screened 35,000 chemicals, searching for those that ignore premature stop signals.

First author Liutao Du developed the screening technology in Gatti's laboratory.

"Of the dozens of active chemicals we discovered, only two were linked to the appearance and function of ATM, the protein missing from the cells of children with A-T," said Du. "These two chemicals also induced the production of dystrophin, a protein that is missing in the cells of mice with a nonsense mutation in the muscular dystrophy gene."

The UCLA team is optimistic that their discovery will aid pharmaceutical companies in creating drugs that correct genetic disorders caused by nonsense mutations. This could affect one in five patients with most genetic diseases, including hundreds of thousands of people suffering from incurable diseases. Because nonsense mutations can lead to cancer, such drugs may also find uses in cancer treatment.

Gatti's lab is funded by the Los Angeles-based Ataxia-Telangiectasia Medical Research Foundation, the National Institutes of Health and the New York-based Ataxia-Telangiectasia Ease Foundation.

The study's coauthors included Robert Damoiseaux, Shareef Nahas, Kun Gao, Hailiang Hu, Julianne Pollard, Jimena Goldstine, Michael Jung, Susan Henning and Carmen Bertoni, all of UCLA.

Elaine Schmidt | EurekAlert!
Further information:
http://www.ucla.edu

More articles from Life Sciences:

nachricht Biofuel produced by microalgae
28.02.2017 | Tokyo Institute of Technology

nachricht Decoding the genome's cryptic language
27.02.2017 | University of California - San Diego

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Safe glide at total engine failure with ELA-inside

On January 15, 2009, Chesley B. Sullenberger was celebrated world-wide: after the two engines had failed due to bird strike, he and his flight crew succeeded after a glide flight with an Airbus A320 in ditching on the Hudson River. All 155 people on board were saved.

On January 15, 2009, Chesley B. Sullenberger was celebrated world-wide: after the two engines had failed due to bird strike, he and his flight crew succeeded...

Im Focus: Breakthrough with a chain of gold atoms

In the field of nanoscience, an international team of physicists with participants from Konstanz has achieved a breakthrough in understanding heat transport

In the field of nanoscience, an international team of physicists with participants from Konstanz has achieved a breakthrough in understanding heat transport

Im Focus: DNA repair: a new letter in the cell alphabet

Results reveal how discoveries may be hidden in scientific “blind spots”

Cells need to repair damaged DNA in our genes to prevent the development of cancer and other diseases. Our cells therefore activate and send “repair-proteins”...

Im Focus: Dresdner scientists print tomorrow’s world

The Fraunhofer IWS Dresden and Technische Universität Dresden inaugurated their jointly operated Center for Additive Manufacturing Dresden (AMCD) with a festive ceremony on February 7, 2017. Scientists from various disciplines perform research on materials, additive manufacturing processes and innovative technologies, which build up components in a layer by layer process. This technology opens up new horizons for component design and combinations of functions. For example during fabrication, electrical conductors and sensors are already able to be additively manufactured into components. They provide information about stress conditions of a product during operation.

The 3D-printing technology, or additive manufacturing as it is often called, has long made the step out of scientific research laboratories into industrial...

Im Focus: Mimicking nature's cellular architectures via 3-D printing

Research offers new level of control over the structure of 3-D printed materials

Nature does amazing things with limited design materials. Grass, for example, can support its own weight, resist strong wind loads, and recover after being...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Booth and panel discussion – The Lindau Nobel Laureate Meetings at the AAAS 2017 Annual Meeting

13.02.2017 | Event News

Complex Loading versus Hidden Reserves

10.02.2017 | Event News

International Conference on Crystal Growth in Freiburg

09.02.2017 | Event News

 
Latest News

Existence of a new quasiparticle demonstrated

28.02.2017 | Materials Sciences

Sustainable ceramics without a kiln

28.02.2017 | Materials Sciences

Biofuel produced by microalgae

28.02.2017 | Life Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>