Navigation Links
Experimental gene therapy treatment for Duchenne muscular dystrophy offers hope for youngster
Date:2/7/2013

Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted -- running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old.

The disease affects about 1 in 3,500 newborns -- mostly boys -- worldwide. It usually becomes apparent in early childhood, as weakened skeletal muscles cause delays in milestones such as sitting and walking. Children usually become wheelchair-dependent during their teens. As heart muscle is increasingly affected, the disease becomes life threatening and many patients die from heart failure in their 20s.

Today, Jacob is one of 51 children participating in a nationwide clinical trial for a new type treatment that could offer help to those suffering from devastating neuromuscular disease. Clinical researchers at UC Davis Medical Center and a handful other research centers around the nation are testing a high-tech drug designed to fix the underlying genetic defect causing the progressive muscular decline that is seen in children with Duchenne.

"This type of genetic therapy is the most exciting treatment approach I have witnessed in my career for Duchenne muscular dystrophy," said Craig McDonald, professor and chair of the Department of Physical Medicine Rehabilitation at UC Davis, as well as principal investigator of the national clinical trial that Jacob is participating in. "We are hopeful that it will delay many of the disease's manifestations and ultimately improve life expectancy for patients."

Duchenne muscular dystrophy is caused by genetic mutations in the gene for the muscle protein dystrophin. The protein is a stabilizer that protects muscle fibers; without enough functional dystrophin, muscles become damaged, causing them to weaken and deteriorate over time.

<
'/>"/>
Contact: Charles Casey
charles.casey@ucdmc.ucdavis.edu
916-734-9048
University of California - Davis Health System
Source:Eurekalert

Page: 1 2 3 4

Related biology news :

1. Experimental drug combination selectively destroys lymphoma cells
2. Nicole George wins 2012 Journal of Experimental Biology Outstanding Paper Prize
3. Compound stimulates tumor-fighting protein in cancer therapy
4. VEGF Inhibition: Therapy-Indicating and Prognostic Biomarkers, 2013
5. Pitt researchers reveal mechanism to halt cancer cell growth, discover potential therapy
6. Changes to DNA on-off switches affect cells ability to repair breaks, respond to chemotherapy
7. BUSM study shows potential of differentiated iPS cells in cell therapy without immune rejection
8. Canada launches first gene therapy trial for Fabry disease
9. New way to kill lymphoma without chemotherapy
10. Gene therapy reprograms scar tissue in damaged hearts into healthy heart muscle
11. New whole plant therapy shows promise as an effective and economical treatment for malaria
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/22/2014)... feature natural habitats and trees and not just asphalt ... to a University of Colorado Boulder study. , Working ... provide stress-reducing benefits for youth, according to a paper ... The study is one of the first of its ... to green settings and stress. , "Many schools already ...
(Date:7/22/2014)... Conn. , July 22, 2014 ... the growing m-commerce market, announces its next generation smart ... 30-minute consumer oriented television show that airs on ... NewsWatch regularly features top technology ... July 31st on the History Channel (6:00-6:30am ET/PT) ...
(Date:7/21/2014)... researchers have detected genetic fragments of Middle East Respiratory ... holding a camel infected with the virus. The work, ... open-access journal of the American Society for Microbiology, indicates ... disease can be transmitted through the air. , MERS, ... has been identified in 699 people as of June ...
Breaking Biology News(10 mins):Natural-terrain schoolyards reduce children's stress, says Colorado University-Boulder study 2Natural-terrain schoolyards reduce children's stress, says Colorado University-Boulder study 3NXT-ID's Next Generation Wocket Smart Wallet to be Featured on NewsWatch, Airing on History Channel, FYI Network and ION Network 2NXT-ID's Next Generation Wocket Smart Wallet to be Featured on NewsWatch, Airing on History Channel, FYI Network and ION Network 3Middle East Respiratory Syndrome coronavirus detected in the air of a Saudi Arabian camel barn 2
... between tiny single-celled algae and highly specialized bacteria. Their ... the oceans by taking nitrogen from the atmosphere and ... use. Details of the symbiosis, published in ... from the investigation of a mysterious nitrogen-fixing microbe with ...
... Just as women are advised to get plenty of folic ... new research suggests another very similar nutrient may one day ... Consuming greater amounts of choline ... may lower an infant,s vulnerability to stress-related illnesses, such as ...
... Bethesda, MD FASEB MARC (Minority Access to Research ... the 2012 Biomedical Engineering Society (BMES) Annual Meeting in ... meant to promote the entry of underrepresented minority students, ... science community and to encourage the participation of young ...
Cached Biology News:Unusual symbiosis discovered in marine microorganisms 2Unusual symbiosis discovered in marine microorganisms 3Unusual symbiosis discovered in marine microorganisms 4Nutrient in eggs and meat may influence gene expression from infancy to adulthood 2Nutrient in eggs and meat may influence gene expression from infancy to adulthood 3
(Date:7/22/2014)... OHAUS Corporation, a leading worldwide ... and scales, proudly announces its Explorer line of ... magazine’s 2014 “Readers' Choice” Award for best ... Explorer was voted the best laboratory balance by ... celebrates “excellence in product design and performance for ...
(Date:7/22/2014)... SHENZHEN, China , 22 juli 2014 /PRNewswire/ ... dochtermaatschappij van BGI, ,s wereld grootste organisatie op het ... een nieuwe service ten behoeve van hele menselijke genoomsequentie ... Complete Genomics. In 2013 acquireerde BGI ... hoog aanzien staat vanwege de in eigen beheer ontwikkelde ...
(Date:7/22/2014)... 22, 2014  Semler Scientific, Inc. (Nasdaq: ... assessment company that develops patented products that assist ... diseases, today announced that it will report second ... U.S. financial markets on July 25, 2014. Doug Murphy-Chutorian, M.D., ... conference call at 11 a.m. EDT, July 25, 2014. ...
(Date:7/22/2014)... Angeles, CA (PRWEB) July 22, 2014 ... test solutions with the debut of its fluorescent ... of Clinical Chemistry (AACC) meeting in Chicago, July ... or bench top versions, the HRDR-300 is a ... pending technology to analyze fluorescent lateral flow immunoassays ...
Breaking Biology Technology:OHAUS Explorer Voted “Best Balance” in Laboratory Equipment’s 2014 Readers’ Choice Awards 2BGI Tech introduceert service op gebied van hele menselijke exoomsequencing op geavanceerd Complete Genomics-platform 2Semler to Report Second Quarter 2014 Financial Results and Host Conference Call on July 25, 2014 2Holomic to Debut HRDR-300 Fluorescent Immunoassay Reader at the AACC 2014 2
... (NYSE: DGX ), the leading provider of diagnostic ... shareholders,on Friday, May 16th at 10:30 am ET., ... Relations,section of the company Web site at http://www.questdiagnostics.com/investor ... broadcast, a replay will be available on,the Web site ...
... study supports the potential for QLT,s drug elution technology ... reduce and ... QLT Inc. (NASDAQ: QLTI; TSX:,QLT) announced today results from ... QLT Plug Delivery, Inc., of its punctal plug,drug delivery ...
... 12 Amylin Pharmaceuticals,Inc. (Nasdaq: AMLN ) will ... May 14, 2008 at 3:05 p.m. CT / 1:05 ... Finance and Chief Financial Officer of,Amylin Pharmaceuticals, will be ... breakout session will be webcast, and a,recording will be ...
Cached Biology Technology:QLT reports initial proof of concept data for punctal plug delivery technology 2QLT reports initial proof of concept data for punctal plug delivery technology 3QLT reports initial proof of concept data for punctal plug delivery technology 4