Application of Gene Correction and Stem Cell Strategies to Neuromuscular Disorders

Unit Code

00131

Department/Institution Offering Unit

Melbourne Neuromuscular Research Unit
St. Vincent's Hospital
41 Victoria Parade
Fitzroy
Victoria 3065 Australia

web: www.mda.org.au (look under "Research")

Unit Points

Research Points: 100

Coursework Points: 0

top of page

Contacts

Unit Coordinator/s:

Dr Robert Kapsa, Dr Andrew Kornberg, Dr Dominic Thyagarajan
Neuromuscular Research Institute
St. Vincent's Hospital
Fitzroy
Victoria 3065 Australia

tel: +61 3 9288 3340 (reception)
fax: +61 3 9288 3350
email: rkapsa@iinet.net.au, kornberg@cryptic.rch.unimelb.edu.au, or thyagad@svhm.org.au

top of page

Overall Objectives

The objective of this program is to give students some exposure to basic laboratory techniques in the area of biochemistry and molecular biology which are at the forefront in the development of cures for hereditary neurological illness. The students would be part of a group of research scientists who are pursuing front-line strategies in the development of cures for major forms of muscular dystrophy, undertaking novel approaches to gene therapy in experimental animals. There would be some exposure to histological and immunocytochemical techniques, as well as basic DNA techniques.

top of page

Content

Coursework: N/A

Research: Research is in the area of developing molecular treatments for hereditary neuromuscular disease. Students will also attend research meetings of the Muscular Dystrophy group held on a weekly basis and will spend one hour a week with one of the co-supervisors.

Sample Research Topics:

1. Involvement in the stem cell transplantation group in the mdx animal model of Duchenne Muscular Dystrophy
2. Development of polyvalent treatment strategies in animal models of Motor Neurone Disease (based in the University of Melbourne Centre for Neuroscience)
3. Studies in disorders of oxidative phosphorylation affecting nerve and muscle (supervisor: Dr Dominic Thyagarajan)
4. Development of Targeted Corrective Gene Conversion Therapies fro Motor Neuron Diseases
5. Stem Cell Therapies for Dystrophic Muscle usiing Non-Muscle Cells
6. Development of Human Gene Correction Therapy for Duchenne Muscular Dystrophy

top of page

Assessment Breakdown

Research Component: 100 points
Research Report (up to 9,000 words): 65%
Supervisor Evaluation: 15%
Oral presentation to senior staff of the Institute: 10%
Written assignment centred around biochemical and molecular advances in the neuromuscular condition being studied (up to 2,000 words): 10%

Coursework Component: 0 points
N/A

top of page

Student Numbers

Number of places available:
3