MRC PhD or MRes/PhD Studentship Molecular and Stem Cell Therapy for Duchenne Muscular Dystrophy



Reference Code: HG050

Closing Date: 14th March 2014

Name of the Supervisors
Professor Hanns Lochm?ller, Institute of Genetic Medicine

Sponsor
Medical Research Council (MRC)

Duration of the Award
Three or four years (optionally an MRes in Neuromuscular Diseases followed by a three-year PhD).

Project Description
Gene therapy and stem cell therapy are molecular approaches that may be applicable to all patients with DMD regardless of their specific mutation once proven effective and safe. In contrast to therapeutic approaches targeting RNA, they also have the potential for permanently restoring dystrophin to muscle following a single application.

Previous work has focussed on identifying suitable stem cells for autologous transplantation including iPS cells (MRC grant with Chris Denning; Dick et al 2011; Dick et all 2013), identifying suitable vector systems and dystrophin constructs for gene transfer into stem cells or into muscle directly (MRC grant with Jenny Morgan; Meng et al, submitted), and establishing tests in vitro and in vivo to assess the benefit of such therapeutic approaches (Jorgensen et al, 2011; M Ritso unpublished; M Reza unpublished). Preliminary results show that dystrophin-deficient, primary cardiomyocytes show a hypertrophy response to stress that can be utilized for the assessment of pharmacological or molecular therapeutic interventions. In addition, we have generated optimized dystrophin constructs that are small enough to be packaged into lentiviral and AAV vectors, but restore nNOS binding to the sarcolemma.

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The project will test which of the nNOS-binding sites are essential, and whether nNOS-restoring dystrophin constructs are superior in restoring function in dystrophin-deficient cardiac and skeletal muscle as compared to conventional micro- or mini-dystrophin molecules. Optimized dystrophin constructs will be tested in both stem cell and gene transfer experiments utilizing the in vitro and in vivo assays mentioned above.

Value of the Award and Eligibility
Depending on how you meet the MRC?s eligibility criteria, you may be entitled to a full or a partial award. A full award covers tuition fees at the UK/EU rate and an annual stipend of ?13,726 (2013/14). A partial award covers fees at the UK/EU rate only.

Person Specification
Candidates should hold or expect to obtain an upper-second-class or higher degree in Biomedical Sciences, Genetics, Biochemistry or a related subject. Those with a Masters qualification in a relevant subject are also encouraged to apply.

How to Apply
You must apply through the university?s online postgraduate application form, via the ‘apply’ button below, selecting ?Master of Research/Doctor of Philosophy (Medical Sciences) Medical Genetics? as the programme of study. Please insert the studentship/partnership reference number HG050. Only mandatory fields need to be completed (no personal statement required) but you must attach a copy of your CV and a covering letter, quoting the title of the studentship and reference number HG050.

Further Information
For further details, please contact:
Dr Steve Laval
E-mail: [email protected]
Telephone: +44 (0) 191 241 8833?



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