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

hin in the participants' muscles the latter results obtained through muscle biopsies at several times during study. Of particular interest to the research teams are the residual effects of the drug several weeks after the injection series is completed.

Although McDonald says it is too early to draw conclusions from the current clinical trial, he suggests there is reason to be optimistic based on animal studies and evidence from another clinical trial in Europe. In that trial, patients and study investigators knew which patients were actually taking the experimental drug, meaning it was an "open" study. The current U.S. study is more rigorous because doctors and families are "blinded" about which drug regimen each participant has -- either one of two dosages of the drug or a placebo -- until the end of the study.

According to McDonald, the antisense oligonucleotide-mediated genetic therapy approach is particularly exciting because of its potential applications to most other variants of Duchenne muscular dystrophy and other genetic diseases. Although the current experimental therapy specifically addresses mutations in only one particular region of the gene, the same therapeutic concept can be applied to many others.

"If successful, this approach can be developed into specific gene therapy that represents truly personalized medicine," said McDonald. "Covering a mutation to restore a normal genetic open reading frame for protein synthesis can be a powerful approach for a variety of genetic diseases."

Standardizing the drug as a therapy for Duchenne patients still requires approval by the U.S. Food and Drug Administration. Investigators say the therapy could be similar to insulin injections for diabetes. But rather than daily injections, the Duchenne treatment might only involve weekly shots, which youngsters could learn to administer on their own.

Now that the clinical trial is closed at UC Davis to new enrollees, McDonald
'/>"/>

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:12/17/2014)... 2014  HITLAB SM announced today its ... to confirm its adherence to current U.S. Food ... HITLAB to conduct regulated smart device and smart ... patient safety and research quality. "HITLAB ... and delivery with innovative technology," said Laura ...
(Date:12/17/2014)... Research and Markets ( http://www.researchandmarkets.com/research/9zq33j/global_chemical ... "Global Chemical Sensor Market 2015-2019" report to ... One major trend upcoming in ... sensors in biomedical applications. Chemical sensors help in ... diagnosis during surgical procedures. The Global Chemical Sensor ...
(Date:12/15/2014)... Research and Markets ( http://www.researchandmarkets.com/research/87pph8/global_facial ... "Global Facial Recognition Market 2015-2019" report to ... Facial recognition is a technology ... system measures the overall facial feature of a ... the distance between eyes. Facial recognition is used ...
Breaking Biology News(10 mins):HITLAB Completes GCP Audit for FDA Smart Device & App Compliance 2Global Chemical Sensor Market 2015-2019: Key Vendors are Abbott Laboratories, Bayer, Hoffmann La-Roche, Johnson & Johnson, NGK Spark Plugs and Robert Bosch 2Global Facial Recognition Market 2015-2019: Key Vendors are 3M Cogent, Cognitec Systems, NEC and Safran Group 2Global Facial Recognition Market 2015-2019: Key Vendors are 3M Cogent, Cognitec Systems, NEC and Safran Group 3
... won the "Most Creative Visualization" award in the "commercial" ... achievement we are extremely proud of. iDEA is Illumina,s ... new and creative visualization and data analysis techniques. ... very first moment we heard about it. Genomatix is ...
... Kan. -- Preliminary research at Kansas State University may make ... scientists believe that cellulose, the most common organic compound on ... biofuels -- if a procedure to effectively break it down ... gives plants their rigidity. Kathrin Schrick, assistant professor in ...
... This press release is available in Spanish ... waters of the European Atlantic Ocean and, nevertheless, resists stock ... the mistakes made by humans. Ms Nerea Goikoetxea, a researcher ... the population dynamics of the northern European hake population, observing ...
Cached Biology News:Learning secrets of world's most common organic compound driving research for biofuels 2Hake population has withstood overfishing, thanks to the warming of the sea 2Hake population has withstood overfishing, thanks to the warming of the sea 3
(Date:12/17/2014)... Bellingham, Washington, USA (PRWEB) December 16, 2014 ... Kirstin Baum, manager at Philipps- Universität Marburg’s Department ... optics and photonics, in the newly released ... 2015-2016. “And never be too shy to ask ... STEM (science, technology, engineering, and mathematics) occupations ranging ...
(Date:12/17/2014)... IN (PRWEB) December 17, 2014 ... Laboratory Excellence following its most recent COLA inspection. ... by COLA, a national healthcare accreditation organization. Accreditation ... standards of quality in day-to-day operations, demonstrate continued ... pass a rigorous on-site laboratory survey. ...
(Date:12/17/2014)... CASI Pharmaceuticals, Inc. (Nasdaq: CASI ... and commercialization of innovative therapeutics addressing cancer and other ... primary commercial focus on China , ... C meeting with the U.S. Food and Drug Administration ... February 2015.  During this meeting the Company will seek ...
(Date:12/15/2014)... 2014 Organovo Holdings, Inc. (NYSE MKT: ... delivering breakthrough 3D bioprinting technology,  today celebrated continued ... its exVive3D TM Human Liver Tissue.  The ... commercial release on November 18, has been recognized ... awards for innovation. Most recently, on ...
Breaking Biology Technology:Network, Multitask, Persevere: SPIE Women in Optics Planner Subjects Share from Experience 2Network, Multitask, Persevere: SPIE Women in Optics Planner Subjects Share from Experience 3Xeno Diagnostics Receives 3rd Award for Laboratory Excellence 2CASI Pharmaceuticals Granted Type C Meeting With U.S. FDA For ENMD-2076 In Fibrolamellar Carcinoma 2CASI Pharmaceuticals Granted Type C Meeting With U.S. FDA For ENMD-2076 In Fibrolamellar Carcinoma 3CASI Pharmaceuticals Granted Type C Meeting With U.S. FDA For ENMD-2076 In Fibrolamellar Carcinoma 4CASI Pharmaceuticals Granted Type C Meeting With U.S. FDA For ENMD-2076 In Fibrolamellar Carcinoma 5Organovo Celebrates a Positive 2014 Reception of its exVive3DTM Human Liver Tissue Product 2Organovo Celebrates a Positive 2014 Reception of its exVive3DTM Human Liver Tissue Product 3
... company developing what it believes to be a novel tool ... tax credits from the State of Wisconsin to ... instead has raised a total of $385,000 from a combination ... to CEO Larry Wells, state law prohibits the company from ...
... Wis. - Last week, the nation marked ... stem cell research. Those restrictions have unquestionably set back ... say. , ,When Congress passed legislation that would ... among families whose loved ones are suffering from a ...
... - After months of speculation, North Prairie Productions, ... selected a site for a biodiesel plant that will produce ... will begin construction on the $42 million facility next spring ... side, where it will relocate its Waterloo offices. , ...
Cached Biology Technology:NovaScan decides against tax credits 2Stem cell research holds the promise for tomorrow 2Stem cell research holds the promise for tomorrow 3Evansville site chosen for biodiesel plant 2