Analysing of utrophin minigenes for functional replacement of dystrophin deficiency using transgenic techniques

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Project objective

There is currently no effective therapy for Duchenne muscular dystrophy (DMD) the most common and severe of the muscular dystrophies affecting approximately 1 in 3000 males. Recent publications on that subject show that one possible therapy to compensate for the lack of dystrophin in patients who suffer from DMD might be the upregulation of the highly homologous autosomal utrophin. This assumption is based on the fact that utrophin is found at the sarcolemma in early human fetal life becomes restricted to the neuromuscular junction once dystrophin is expressed. In the mdx mouse, the animal model of DME utrophin expression is high in muscle at birth but declines during the first weeks of life, during which time the animal begin to develop myonecrotic lesions. It will be challenging to examine whether utrophin can replace dystrophin in vivo using transgenic techniques. Therefore, the outlined proposal will focus on the production and detailed analysis of mdx mice which . transgenic for the utrophin gene under the control of a strong muscle promoter. Training content (objective, benefit and expected impact) Prof. Davies group at the Department of Biochemistry at the Oxford University is one of the leading laboratories in the research field of muscular genetic disorders with a very high international reputation. The proposal has been formulated in collaboration with Prof. Davies. The Oxford group can provide excellent expertise and experience in all technologies necessary to carry out this project successfully. Therefore I can aquire a lot of modern methods widely applicable in the research fields of molecular genetics, biochemistry and cell biology. In summary, I regard the facilities and resources (libraries, courses and seminars) available at the University of Oxford most beneficial for my independent scientific endeavors.

Original text from CORDIS.

Participants

• THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD · OXFORDCoordinatorUnited Kingdom
• Not availableCity levelAustria

Links

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