Navigation Links
No small feat: First ever gene therapy success for muscular dystrophy achieved

Researchers from the University of Pittsburgh report the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable scientific challenges that have cast doubt on gene therapy ever being feasible for children with muscular dystrophy can be overcome. Moreover, their results, published in this week's online edition of the Proceedings of the National Academy of Sciences (PNAS), indicate that a single treatment can have expansive reach to muscles throughout the body and significantly increase survival.

CMD is a group of some 20 inherited muscular dystrophies characterized by progressive and severe muscle wasting and weakness first noticed soon after birth. No effective treatments exist and children usually die quite young.

Despite gene therapy being among the most vigorously studied approaches for muscular dystrophy, it has been beset with uniquely difficult hurdles. The genes to replace those that are defective in CMD are larger than most, so it has not been possible to apply the same methods successfully used for delivering other types of genes. And because CMD affects all muscles, an organ that accounts for 40 percent of body weight, gene therapy can only have real therapeutic benefit if it is able to reverse genetic defects in every cell of the body's 600 muscle groups.

By using a miniature gene, similar in function to the one defective in CMD, and applying a newly developed method for "systemic" gene delivery, the Pitt researchers have shown that gene therapy for muscular dystrophy is both feasible and effective in a mouse model of especially profound disease. Using this approach, the team, led by Xiao Xiao, Ph.D., associate professor of orthopaedic surgery and molecular genetics and biochemistry at the University of Pittsburgh School of Medicine, report that treated mice had physiological improvements in the muscles of the heart, diaphragm, abdomen and l
'"/>

Source:University of Pittsburgh Medical Center


Page: 1 2 3

Related biology news :

1. Stopping smallpox in its tracks: A new anti-viral approach
2. New World founders small in number
3. Secret of smallpoxs success may lead to bioterror cure
4. Circulating stem cells play small role in lung repair
5. LIAI scientists make major finding on potential smallpox treatment
6. Researchers develop new testing methods for potential monkeypox or smallpox outbreak
7. Solexa and collaborating scientists illuminate the small RNA component of the transcriptome
8. Delaware scientists make significant advance in study of small RNAs
9. Scientists learn to predict protein-stabilizing ability of small molecules
10. One small step means giant leap for spinal cord research
11. New study of the worlds smallest elephant
Post Your Comments:
(Date:5/17/2013)... for Experimental Biology (FASEB) released a new analysis ... highlighting the devastating impact of sequestration on the nation,s ... a May 15th Senate Appropriations Committee hearing featuring NIH ... $1.6 billion cut the agency sustained in FY 2013 ... the scientific community." , According to data from ...
(Date:5/16/2013)... HOUSTON African-American adults living closer to a fast ... than those who lived further away from fast food, ... Anderson Cancer Center, and this association was particularly strong ... new study published online in the American Journal ... residential proximity to a fast food restaurant, and among ...
(Date:5/16/2013)... May 16, 2013 The relationship between the ... has not been clear. Schizophrenia is commonly associated ... There are clues that reduced IQ may be ... example, reduced cognitive ability may precede the onset ... present in healthy relatives of people diagnosed with ...
Breaking Biology News(10 mins):Body mass index of low income African-Americans linked to proximity of fast food restaurants 2Body mass index of low income African-Americans linked to proximity of fast food restaurants 3Genetic risk for schizophrenia is connected to reduced IQ 2
... groundbreaking environmental study to be published in a prestigious ... end up in fish in as little as three ... Canada, played a key role in designing and carrying ... mercury emissions from coal-fired power plants and other industrial ...
... WEST LAFAYETTE, Ind. -- Biologists have determined the three-dimensional ... in the assembly of new viruses. The Paramecium ... transferring its DNA into host cells. Once inside the ... which is needed to produce structural proteins that are ...
... mining evidence from the classic films made by Mitchell ... suffered something of a setback in Victorian England, at ... times, lefties, numbers quickly rose again, the researchers report ... of Cell Press. , "Left-handedness is important because ...
Cached Biology News:Scientists learn structure of enzyme in unusual virus 2Films of Mitchell and Kenyon illuminate lefties' decline in Victorian England 2
(Date:5/16/2013)... (PRWEB) May 16, 2013 Cambridge Healthtech ... from August 19-23, 2013 at the Renaissance Waterfront Hotel ... Summit provide a close-up look at the current strategies ... ensuring bottom-line quality. Leaders in the field will ... to produce biopharmaceuticals with greater specificity and efficiency. , ...
(Date:5/16/2013)... , May 16, 2013 Scientists at the ... Beijing , and the California ... demonstrated a novel therapy for reversing memory decline in ... they find significant cognitive improvement in advanced stage AD ... mechanism for reversing cognitive decline for advanced stage AD ...
(Date:5/16/2013)... , May 16, 2013 /PRNewswire/ ... market research report is available in ... Diagnostic Cardiac Biomarkers for Acute Coronary ... Forecasts http://www.reportlinker.com/p01182969/MediPoint-Diagnostic-Cardiac-Biomarkers-for-Acute-Coronary-Syndromes---APAC-Analysis-and-Market-Forecasts.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=In_Vitro_Diagnostic ... for Acute Coronary Syndromes - APAC ...
(Date:5/16/2013)... 2013 A number of residents in an ... a report from abcactionnews.com, it was no thanks to the ... Cleaning Products recommended to the management and everyone else suffering ... also encouraged the public to try samples of it to ... dated May 11, 2013 determined that the complex ...
Breaking Biology Technology:The 2013 Bioprocessing Summit to Bring Together International Leaders to Discuss Today's Bioprocess Issues From Cell Line Selection to Manufacturing in Boston, MA 2Elevation of Brain Magnesium Reverses Memory Deficits in Alzheimer Mice 2Elevation of Brain Magnesium Reverses Memory Deficits in Alzheimer Mice 3MediPoint: Diagnostic Cardiac Biomarkers for Acute Coronary Syndromes - APAC Analysis and Market Forecasts 2MediPoint: Diagnostic Cardiac Biomarkers for Acute Coronary Syndromes - APAC Analysis and Market Forecasts 3MediPoint: Diagnostic Cardiac Biomarkers for Acute Coronary Syndromes - APAC Analysis and Market Forecasts 4MediPoint: Diagnostic Cardiac Biomarkers for Acute Coronary Syndromes - APAC Analysis and Market Forecasts 5MediPoint: Diagnostic Cardiac Biomarkers for Acute Coronary Syndromes - APAC Analysis and Market Forecasts 6MediPoint: Diagnostic Cardiac Biomarkers for Acute Coronary Syndromes - APAC Analysis and Market Forecasts 7MediPoint: Diagnostic Cardiac Biomarkers for Acute Coronary Syndromes - APAC Analysis and Market Forecasts 8Bed Bugs Plague Tarpon Springs Apartment Complex, Non-Pesticide Bed Bug Spray Recommended to Management by My Cleaning Products to Help 2Bed Bugs Plague Tarpon Springs Apartment Complex, Non-Pesticide Bed Bug Spray Recommended to Management by My Cleaning Products to Help 3
... as 1 cell , Karen Dolter Jeff ... microprep kit provides a,convenient method for isolating total RNA ... bound to a solid support, which eliminates the,need for ... quality, with no detectable DNA contamination and is suitable ...
... transduction pathway of your choice , ... Cloning Systems, Inc. , The PathDetect in ... for assessing the in vivo activation of ... a unique fusion trans-activator,plasmid that expresses pathway-specific ...
... , Eagle Eye Optical Filter Optimized for ... A new optical filter has been developed for the Eagle ... increases contrast and sensitivity when imaging,silver-stained protein gels. , The ... currently sold with an optical filter optimized for imaging nucleic ...
Cached Biology Technology:Fast Isolation of Total RNA from Small Numbers of Cells 2Fast Isolation of Total RNA from Small Numbers of Cells 3Fast Isolation of Total RNA from Small Numbers of Cells 4Fast Isolation of Total RNA from Small Numbers of Cells 5Fast Isolation of Total RNA from Small Numbers of Cells 6New Fusion Trans-Activator Plasmids for Studying Signal Transduction,Pathways 2New Fusion Trans-Activator Plasmids for Studying Signal Transduction,Pathways 3New Fusion Trans-Activator Plasmids for Studying Signal Transduction,Pathways 4New Fusion Trans-Activator Plasmids for Studying Signal Transduction,Pathways 5New Fusion Trans-Activator Plasmids for Studying Signal Transduction,Pathways 6Eagle Eye Optical Filter Optimized for Imaging Silver-Stained,Gels 2Eagle Eye Optical Filter Optimized for Imaging Silver-Stained,Gels 3
MOUSE ANTI HUMAN MBP (67-74) Immunogen: Human myelin basic protein...
NORMAL DONOR DONKEY SERUM...
... Probes Fc OxyBURST Green reagent (F-2902) was ... Boston University to permit measurement of the ... the subsequent oxidative burst directly in the ... consists of bovine serum albumin (BSA) that ...
Because it is manufactured at our ISO 9001–certified facilities in Eugene, Oregon, we can guarantee that FluoroPure grade propidium iodide is greater than or equal to 98% pure by HPLC....
Biology Products: