Navigation Links
LSUHSC research discovery provides therapeutic target for ALS
Date:12/19/2012

New Orleans, LA Research led by Dr. Udai Pandey, Assistant Professor of Genetics at LSU Health Sciences Center New Orleans, has found that the ability of a protein made by a gene called FUS to bind to RNA is essential to the development of Amyotrophic Lateral Sclerosis (ALS). This discovery identifies a possible therapeutic target for the fatal neurological disease. The research will be available online in the Advanced Access section of the journal Human Molecular Genetics website, posted by December 21, 2012. It will be published in an upcoming issue of the journal.

The current project advances Dr. Pandey's ALS research by teasing out specifically how the FUS gene causes the disease. To find out whether or not the RNA binding ability of FUS was required for the disease pathogenesis, the researchers mutated FUS RNA binding sites and produced a version of FUS that couldn't bind RNA, both with and without ALS mutations. They found that not only could they eliminate FUS RNA binding, but when they blocked RNA binding, they also suppressed ALS related neurodegeneration, demonstrating that the RNA binding ability of FUS is essential to the ALS disease process.

The researchers are working with fruit flies the first animal model of FUS-related ALS, a model Dr. Pandey developed. The fruit flies were engineered to carry and express a mutated human FUS gene. This mutated FUS gene has been shown to be one of the causes of both familial and sporadic ALS. In the fruit flies, the resulting neurodegeneration impairs their ability to walk or climb and the defect is also easily visualized in the structure of their eyes. In addition, the flies carrying the defective FUS gene demonstrate hallmarks of the human disease, such as an age-dependent degeneration of neurons, accumulation of abnormal proteins and a decrease in life span. The fly model is a valuable resource for performing drug screens to identify drugs that could modify the effects of the mutated gene in humans.

"Our findings may pave the way for development of drugs targeting the biological processes responsible for causing ALS, and leading to treatments or prevention of this currently fatal, incurable condition, " notes Pandey. "The fly model is an inexpensive and fast way to study ALS as well as many human diseases such as cancer, Alzheimer's disease and Parkinson's disease. Many basic biological processes are well conserved between humans and fruit flies, and nearly 75% of human disease-causing genes are believed to have a functional partner (homolog) in the fly that makes these small animals a highly tractable model system."

"These intriguing findings inspire us and other researchers to search for drugs that can make the defective FUS protein less toxic by targeting is RNA binding as a potential therapeutic intervention," noted Gavin Daigle (Graduate student in the Pandey lab and leading author of the manuscript).

According to the National Institutes of Health, Amyotrophic Lateral Sclerosis, sometimes called Lou Gehrig's disease, is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons. Motor neurons are nerve cells located in the brain, brainstem, and spinal cord that serve as controlling units and vital communication links between the nervous system and the voluntary muscles of the body. Messages from motor neurons in the brain (called upper motor neurons) are transmitted to motor neurons in the spinal cord (called lower motor neurons) and from them to particular muscles. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die, ceasing to send messages to muscles. Unable to function, the muscles gradually weaken, waste away (atrophy), and twitch (fasciculations). Eventually, the ability of the brain to start and control voluntary movement is lost.


'/>"/>

Contact: Leslie Capo
lcapo@lsuhsc.edu
504-568-4806
Louisiana State University Health Sciences Center
Source:Eurekalert

Related biology news :

1. LSUHSCs Weiss chosen to help set national eye policy, research
2. LSUHSC research identifies new therapeutic target for Alzheimers disease
3. LSUHSC research finds ginkgo biloba doesn’t improve cognitive function in MS
4. LSUHSC research finds treating stress prevented new MS brain lesions
5. LSUHSC research finds HPV-related head & neck cancers rising, highest in middle-aged white men
6. Scale-up of a temporary bioartificial liver support system described in BioResearch Open Access
7. Desert Research Institute utilizing IBM big data analytics to assist Nevadas growth strategy
8. CNIO researchers develop new databases for understanding the human genome
9. New research predicts rising trend in Indias Violent Land Conflicts; 130 districts struggle
10. 3 Johns Hopkins researchers named AAAS Fellows
11. The Sackler Institute unveils a global research agenda for nutrition science
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/2/2016)... Feb. 2, 2016 This BCC Research ... market by reviewing the recent advances in high ... drive the field forward. Includes forecast through 2019. ... the challenges and opportunities that exist in the ... solution developers, as well as IT and bioinformatics ...
(Date:2/1/2016)... , February 1, 2016 ... advancements to drive global touchfree intuitive gesture control market ... --> Rising sales of consumer electronics coupled with ... control market size through 2020 ... electronics coupled with new technological advancements to drive global ...
(Date:1/25/2016)...   Unisys Corporation (NYSE: UIS ) today announced ... International Airport, New York City , to help ... to enter the United States using passports ... pilot testing of the system at Dulles last year. The ... during January 2016. --> pilot testing of the ...
Breaking Biology News(10 mins):
(Date:2/11/2016)... International, a not-for-profit organization focused on the ethics and governance ... to patients around the world, today announced that the editors ... the Good Pharma Scorecard an ,Editors, Pick, ... of BMJ Open ,s ,Most Popular Articles, which includes ... read. Ed Sucksmith , assistant editor of ...
(Date:2/11/2016)... BEACH, Florida , February 11, 2016 /PRNewswire/ ... PositiveID Corporation ("PositiveID" or "Company") (OTCQB: PSID), ... diagnostics, announced today that its Thermomedics subsidiary, which ... progress on its growth plan in January 2016, ... products distributors, increasing sequential monthly sales growth, and ...
(Date:2/10/2016)... MONTREAL , Febr. 10, 2016 /PRNewswire/ - BioAmber Inc. ... is pleased to announce that Mitsui & Co. Ltd., ... bio-based succinic acid plant, is investing an additional CDN$25 ... equity, increasing its stake from 30% to 40%.  Mitsui ... of bio-succinic acid produced in Sarnia ...
(Date:2/10/2016)... York, New York (PRWEB) , ... ... ... Pharmaceuticals Inc. (NASDAQ: REGN) today announced that it has joined the Human ... and immunotherapies for infectious diseases and cancer. , The Human Vaccines ...
Breaking Biology Technology: