Navigation Links
UCLA study identifies 2 chemicals that could lead to new drugs for genetic disorders
Date:9/28/2009

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.


'/>"/>

Contact: Elaine Schmidt
eschmidt@mednet.ucla.edu
310-794-2272
University of California - Los Angeles
Source:Eurekalert

Related medicine news :

1. New study resolves the mysterious origin of Merkel cells
2. Many Kids Suffer Medication Side Effects: Study
3. Swiss study finds income affects prostate cancer patients survival
4. Study finds intervention program increases kids healthy eating, reduces screen time
5. Exercise May Prevent Prostate Cancer: Study
6. Video: Despite Increased Health Threat, New Study Finds Low Concern About Flu Among Hispanics
7. New England Journal of Medicine Also Publishes Mayo Clinic Study of Physicians Beliefs About Health Care Reform
8. New England Journal of Medicine publishes Mayo Clinic study about health care reform
9. HIV vaccine regimen demonstrates modest preventive effect in Thailand clinical study
10. Volcano Corporation Praises FAME Study Results
11. Study reveals 2/3 of prostate cancer patients do not need treatment
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/26/2016)... (PRWEB) , ... June 26, 2016 , ... Pixel Film ... Pro X. , "Film editors can give their videos a whole new perspective by ... Austin - CEO of Pixel Film Studios. , ProSlice Levels contains over 30 ...
(Date:6/25/2016)... ... June 25, 2016 , ... Experts from the American ... Annual Research Meeting June 26-28, 2016, at the Hynes Convention Center in Boston. ... including advance care planning, healthcare costs and patient and family engagement. , AIR ...
(Date:6/25/2016)... ... June 25, 2016 , ... First Choice Emergency Room , the largest ... as the Medical Director of its new Mesquite-Samuell Farm facility. , “We are ... Mesquite location,” said Dr. James M. Muzzarelli, Executive Medical Director of First Choice Emergency ...
(Date:6/25/2016)... ... , ... Conventional wisdom preaches the benefits of moderation, whether it’s a matter ... bar too high can result in disappointment, perhaps even self-loathing. However, those who set ... , Research from PsychTests.com reveals that behind the tendency to set low ...
(Date:6/24/2016)... , ... June 24, 2016 , ... Marcy was in a crisis. Her son James, ... out at his family verbally and physically. , “When something upset him, he couldn’t control ... use it. He would throw rocks at my other children and say he was going ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... Research and Markets has announced the ... to 2022" report to their offering. ... patients with kidney failure, it replaces the function of kidneys ... blood and thus the treatment helps to keep the patient ... Increasing number of ESRD patients & substantial ...
(Date:6/23/2016)... , June 23, 2016 ... "Pharmaceutical Excipients Market by Type (Organic Chemical (Sugar, ... Formulation (Oral, Topical, Coating, Parenteral) - Global Forecast to ... The global pharmaceutical excipients market ... at a CAGR of 6.1% in the forecast period ...
(Date:6/23/2016)... 23, 2016 Roche (SIX: RO, ROG; OTCQX: ... its Elecsys BRAHMS PCT (procalcitonin) assay as a dedicated ... shock. With this clearance, Roche is the first IVD ... solution for sepsis risk assessment and management. ... and PCT levels in blood can aid clinicians in ...
Breaking Medicine Technology: