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:10/14/2014)... discriminating thieves, prostate cancer tumors scavenge and hoard copper ... such avarice may be a fatal weakness. , ... kill prostate cancer cells by delivering a trove of ... diseased cells brimming with the mineral, leaving non-cancer cells ... already commercially available for other uses, could soon be ...
(Date:10/14/2014)... the planet,s leading questions is how to produce enough ... climate. The Food and Agriculture Organization of the United ... the next 40 years to feed a growing global ... necessary rise in food production. Plants—grains, cereals, fruits, ... livestock. Current research must tap into our knowledge ...
(Date:10/14/2014)... led by researchers at the University of Massachusetts ... Miller School of Medicine (UMMSM) have identified what ... (manic depressive) disorder, a breakthrough that could lead ... as well as depression and other related mood ... week in Nature Molecular Psychiatry , show ...
Breaking Biology News(10 mins):Prostate cancer's penchant for copper may be a fatal flaw 2Building a bridge from basic botany to applied agriculture 2Building a bridge from basic botany to applied agriculture 3Rare genetic disease protects against bipolar disorder 2Rare genetic disease protects against bipolar disorder 3
... Engineering Conferences International (ECI) ( info@engconfintl.org ... XII Conference ( http://www.engconfintl.org/10ac.html ) are proud ... the winner of the 2010 Cell Culture ... Professor of Chemical and Biomolecular Engineering, at ...
... the Iberian Peninsula usually exceed the protection threshold established by ... according to data gathered between 2000 and 2005 by four ... Doana National Park. The limits established to protect human ... usually exceeded from April onwards, especially during the summer, in ...
... Langone Medical Center researchers have developed a powerful new method ... genes and examine how the process goes wrong in cancer ... research and described in the April 9 issue of ... DNA, a process required for gene activation. "The ...
Cached Biology News:Michael Betenbaugh wins 2010 Cell Culture Engineering award 2Ozone levels tend to be above recommended levels at this time of year in South West Spain 2Powerful new method allows scientists to probe gene activation 2
(Date:10/19/2014)... The Latin American hardware encryption display market report defines ... and forecast of revenue. This market was valued at ... $2,366.8 million by 2018, at a CAGR of 70.3% ... of the Latin American hardware encryption market report to ... also provides a glimpse of the segmentation of the ...
(Date:10/19/2014)... 2014 The Asia-Pacific Bromine Market ... Asia-Pacific with analysis and forecast of revenue. , ... Bromine Market report, to get an idea of ... glimpse of the segmentation in the Asia-Pacific bromine ... figures. , http://www.micromarketmonitor.com/market/asia-pacific-bromine-6741503144.html , Bromine ...
(Date:10/19/2014)... October 19, 2014 The report "Chocolate, ... Share, Trade, Prices, Geography Trend and Forecast (2011 - ... category, and geography and studies the major market drivers, ... America, Europe, and Asia. , The global chocolate market ... $98.3 billion in 2016 at an estimated CAGR of ...
(Date:10/19/2014)... According to market research report ... Controllers; Wireless gateways)]- Global Advancements, Forecasts and Analysis ... Outdoor Wi-Fi market with analysis and forecasting of ... identifies the drivers and the restrains for this ... and challenges. , Browse 65 market data tables ...
Breaking Biology Technology:The Latin American hardware encryption display market is expected to reach $2,366.8 million by 2018 - New Report by MicroMarket Monitor 2The Latin American hardware encryption display market is expected to reach $2,366.8 million by 2018 - New Report by MicroMarket Monitor 3The Asia-Pacific Bromine Market is estimated to grow to $4,080.1 million by 2018 - New Report by MicroMarket Monitor 2The Asia-Pacific Bromine Market is estimated to grow to $4,080.1 million by 2018 - New Report by MicroMarket Monitor 3Chocolate Market Projected To Reach $98.3 Billion by 2016 - New Report by MarketsandMarkets 2Chocolate Market Projected To Reach $98.3 Billion by 2016 - New Report by MarketsandMarkets 3Chocolate Market Projected To Reach $98.3 Billion by 2016 - New Report by MarketsandMarkets 4Outdoor Wi-Fi Market (by Access Points, WLAN Controllers, Wireless gateways) Expected to Cross $37.2 Billion by 2018 - New Report by MarketsandMarkets 2Outdoor Wi-Fi Market (by Access Points, WLAN Controllers, Wireless gateways) Expected to Cross $37.2 Billion by 2018 - New Report by MarketsandMarkets 3Outdoor Wi-Fi Market (by Access Points, WLAN Controllers, Wireless gateways) Expected to Cross $37.2 Billion by 2018 - New Report by MarketsandMarkets 4
... Acquisition Provides Expanded Services to the Pharmaceutical and Biotech ... ... ANN ARBOR, Mich. and KIRKBYMOORSIDE, England, Sept. 10 /PRNewswire-,USNewswire/ ... public health protection, today,announced the acquisition of David Begg Associates ...
... BRIDGEWATER, Mass., Sept. 10 Pressure,BioSciences, Inc. (Nasdaq: ... the advantages of pressure cycling technology (PCT) in ... (2) detect,biomarkers of colon cancer, and (3) detect ... this week in three presentations at the 29th,Annual ...
... Development on the ... BURLINGTON, Mass., Sept. 10 Hyaluron Contract,Manufacturing (HCM), a ... pharmaceutical and biotechnology industries, announced,today that it has been ... Impact Award. HCM is one of five companies from ...
Cached Biology Technology:NSF International Acquires David Begg Associates 2Three Studies by Independent Scientists Highlighting Pressure Cycling Technology (PCT) to be Presented this Week at the British Mass Spectrometry Society's 29th Annual Meeting 2Three Studies by Independent Scientists Highlighting Pressure Cycling Technology (PCT) to be Presented this Week at the British Mass Spectrometry Society's 29th Annual Meeting 3Hyaluron Contract Manufacturing Named a 2007 Economic Impact Award Finalist 2
... 220-240 V, is a microfluidics-based system that ... including some data analysis, in 30 minutes. ... analysis (it does not include the vortex ... includes the electrophoresis station, priming station, software, ...
...
... originally formulated to support the growth of ... moth,Antherea eucalypti. The medium is a modification ... The cell lines established by Grace using ... developed. The basal medium, when properly supplemented, ...
... Programmable and Digital Orbital Shaker. Our durable, rugged ... make every day use even easier. This advanced ... to 4 different programs. Its easy recall function ... the VSOS-4P has a pulse operation for a ...
Biology Products: