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:10/16/2014)... war on the human body. Battles are won, ... pancreatic cancer, this stalemate—known as tumor dormancy—can last ... a phenomena that is poorly understood. , ... of Salvatore Torquato, a Professor of Chemistry at ... tumor dormancy and the switch to a malignant ...
(Date:10/16/2014)... thinking on how the bowel lining develops and, in ... cancer starts. , The researchers produced evidence that stem ... that are a feature of the bowel lining, and ... development, a controversial finding as scientists are still divided ... technologies, Dr Chin Wee Tan and Professor Tony Burgess ...
(Date:10/16/2014)... Calif. (October 16, 2014) – Post-menopausal women experienced ... on hormone levels or genital bleeding, after 12 ... an investigational fermented soy germ-based nutritional supplement previously ... to a new peer-reviewed pilot study reported in ... (NAMS) annual scientific meeting. , "These data ...
Breaking Biology News(10 mins):Modeling tumor dormancy 2Cryptic clues drive new theory of bowel cancer development 2S-equol supplements associated with improved measures of reproductive health in postmenopausal women 2
... Grand Canyon may be so old that dinosaurs once lumbered ... the University of Colorado at Boulder and the California Institute ... radiometric dating to show the Grand Canyon may have formed ... origins by 40 million to 50 million years. The ...
... researchers is needed to make progress towards one ... marks contribute to regulation of gene expression. This ... European Science Foundation (ESF), Computational Approaches to the ... Epigenetics studies features of the DNA and chromatin ...
... NEW YORK, April 9 e-Smart(R) Technologies,Inc. ... and its parent,company, IVI Smart Technologies, Inc., ... filed in a Motion last month by,IDsmart ... Northern District of,California, San Francisco Division, Case ...
Cached Biology News:Grand Canyon may be as old as dinosaurs, says new study 2Grand Canyon may be as old as dinosaurs, says new study 3Computation to unravel how genes are regulated and shed light on how cells become different 2Computation to unravel how genes are regulated and shed light on how cells become different 3IDsmart Admits in Court Document That e-Smart's Technology was Used to Develop IDsmart's Smart Card 2IDsmart Admits in Court Document That e-Smart's Technology was Used to Develop IDsmart's Smart Card 3
(Date:10/25/2014)... Oct 24, 2014 Research and Markets ... Market by Product , Research Area & by End ... their offering. In this report, the ... products, research areas, and end users. The product segments ... enzymes, instruments & consumables, kits, and reagents. The subsegments ...
(Date:10/22/2014)... October 21, 2014 The Americas ... inorganic refrigerants market in Americas with analysis and ... of the Americas Inorganic Refrigerants Market report, to ... This also provides a glimpse of the segmentation ... supported by various tables and figures. , ...
(Date:10/22/2014)... Diego, CA (PRWEB) October 22, 2014 ... balances from Sartorius, A & D Weighing, Rice ... Sartorius CPA Semi-Micro Balance . The Sartorius CPA ... for an affordable high-quality, precise, and user-friendly laboratory ... manufacturing of laboratory equipment, their laboratory balances are ...
(Date:10/22/2014)... Oct. 22, 2014 /PRNewswire/ - iCo Therapeutics ("iCo" or ... next steps for its Oral Amphotericin B program.  ... in vitro work involving samples from ... plans to complete pre clinical studies and regulatory ... 1A clinical trial, utilizing approximately $700,000 of funding ...
Breaking Biology Technology:Global Epigenetics (VLA, WBA, NBA, RTA, Rotary-Wing Aircraft) Market - Forecast to 2019 2Global Epigenetics (VLA, WBA, NBA, RTA, Rotary-Wing Aircraft) Market - Forecast to 2019 3The Americas Inorganic Refrigerants Market is estimated to grow to $71.6 million by 2018 - New Report by MicroMarket Monitor 2The Americas Inorganic Refrigerants Market is estimated to grow to $71.6 million by 2018 - New Report by MicroMarket Monitor 3Pipette.com Launches the Addition of Sartorius CPA Semi-Micro Balance to Their Product Portfolio with Special Promotional Pricing 2Pipette.com Launches the Addition of Sartorius CPA Semi-Micro Balance to Their Product Portfolio with Special Promotional Pricing 3iCo Therapeutics Announces Advancement of Oral Amphotericin B Program 2iCo Therapeutics Announces Advancement of Oral Amphotericin B Program 3
... July 10 CV Therapeutics,Inc. (Nasdaq: CVTX ) ... European Medicines Agency (EMEA) for ranolazine for,the treatment of ... (EU),member states. This approval follows a positive opinion from ... on April 24, 2008., Ranolazine is approved for ...
... MADISON, N.J., July 10 Quest Diagnostics,Incorporated (NYSE: ... and services, announced today that its second-quarter,2008 results will ... market,opens, and that it will hold its quarterly conference ... a.m. ET on that day., The public may ...
... Inc. (Nasdaq: ALXN ), today announced that the ... on Tuesday, July 29, 2008, at,10:00 a.m. Eastern Time ... for the second quarter ended June 30, 2008. Alexion,anticipates ... Tuesday,July 29, 2008., To participate in this conference ...
Cached Biology Technology:CV Therapeutics Receives European Marketing Authorization for Ranolazine for the Treatment of Patients With Chronic Angina 2CV Therapeutics Receives European Marketing Authorization for Ranolazine for the Treatment of Patients With Chronic Angina 3Quest Diagnostics to Release Second Quarter 2008 Financial Results 2
Ephrin A4...
Rad51B...
... Rabbit polyclonal to Ki67 - Proliferation Marker, ... cell cycle related nuclear protein, expressed by ... active cell cycle (G1, S, G2 and ... (G0) cells. Ki67 antibodies are useful in ...
... reactive against the complex IV assembly factor SURF1. ... cultured cells. SURF1 is an important assembly factor ... participate in the folding of subunit II and ... SURF1, particularly ones that result in an absence ...
Biology Products: