�Research Scientists at Oxford University suffer received �150k raised by Action Duchenne to fund the in style research into 'Exon skipping', a gene therapy approach to deal Duchenne Muscular Dystrophy. The project forms part of the bigger research plan currently beingness undertaken by MDEX Consortium* UK, a group of research scientists that has received all over �2M of funding from the UK government and Medical Research Council (MRC) to convey clinical trials on DMD patients.
Duchenne affects 2,500 citizenry in the UK and about 40,000 citizenry worldwide. It has no cure and is caused by mutations in the dystrophin cistron that prevent correct indication of the genetic code, the resultant of which is that the essential muscle protein dystrophin is not produced. This results in progressive muscle wasting, severely limiting mobility in teenage patients (boys and men) and leading to premature dying in their twenties from respiratory or cardiac problems (the heart muscle is directly affected by the genetic mar). The aim of any therapy to cure DMD is to restore dystrophin protein to the affected muscle and heart tissues.
Recent research by the MDEX Consortium has demonstrated that short molecules - called antisense oligonucleotides (AOs) - bottom restore the production of dystrophin protein to affected tissues. This is through by a process called 'exon skipping', in which the parts of the genetic code affected by a genetic mutation are "patched" AO's so that that the code can be correctly read.
This a la mode research project is existence headed up by Matthew Wood at Oxford University, a member of the MDEX Consortium**: "This exciting new inquiry project is made possible by Action Duchenne and its donation of support. As a result of current advancement with AO technology we believe that we ar now on the verge of having a inherited treatment for Duchenne and of beingness able to offer tangible hope to Duchenne sufferers and their families."
In clinical trials to escort AO legal transfer - regular with injections of high doses - has been poor, since the tier of dystrophin production is not high school enough in muscle cells nor is it arrival the heart. Wood's research will look into further slipway to amend the delivery of AOs by the use of different types of AO chemistry (including so-called peptide nucleic acids or PNAs) and by attaching short sections of cell penetrating peptides which dramatically meliorate AO delivery to all muscle groups, including the heart. The Wood lab in Oxford is functional closely with Dr Mike Gait and colleagues at the MRC Laboratory of Molecular Biology in Cambridge who have also been developing newfangled cell acute peptides as PNA conjugates for enhanced delivery into cells.
Nick Catlin, CEO of Action Duchenne aforesaid; "This research project is groundbreaking in the treatment of Duchenne. While Exon skipping will not be a finish cure for Duchenne brawny dystrophy, it will be a therapy and should reduce the severity of its symptoms. It is therefore imperative that further funds are found to help solve the leftover problems with developing these medicines and to get to them available as shortly and as widely as possible to all Duchenne sufferers."
*The MDEX Consortium is funded by the department of Health and groups together nine researchers as well as the charities Muscular Dystrophy Campaign, Action Duchenne and Duchenne Parents Support Group.
**This genetic engineering may as well be applied in the research for diseases such as crab and the HIV virus, where the particular factor can be disabled, causation the malignant cells to die.
About Action Duchenne
Action Duchenne (officially Parent Project UK) was set up by Duchenne families in 2001 to promote new research for a cure for Duchenne. The polemonium caeruleum has a strong record in backing research and has to date funded 8 major projects costing �800,000 and has lead the �1.6m DoH financing of the MDEX project. These projects have enabled much requisite early work to be completed on exon skipping and other therapeutic approaches.
Action Duchenne holds an international conference every year to bring together researchers and families to exchange new research developments and provide a vital coming together venue for scientists.
In 2005 Action Duchenne launched the Duchenne Registry, the first National Duchenne database that holds gene information of people living with Duchenne and can be used to speed up the recruitment of patients for clinical trials.
In 2006 Action Duchenne launched a comprehensive learning and behaviour toolkit for manipulation by parents and education Department professionals.
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