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PhD Research Project

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and may not be available.


Gene therapy for muscular dystrophy, atherosclerosis & HIV

Dept/School/Faculty:
PhD Supervisor:
Application Deadline:
Applications accepted all year round
Funding Availability:
Self-Funded PhD Students Only

The activities in the Dickson lab involve development of pre-clinical gene therapies for cardiovascular, neuromuscular & infectious diseases, and the engineering and testing of viral (adenovirus, AAV, lentivirus) and non-viral (plasmid, oligonucleotide) gene therapy vectors in pre-clinical models in the areas of Duchenne muscular dystrophy, atherosclerosis, and HIV. Research will involve vector design, engineering and production, and evaluation of therapeutic efficacy in cell culture and transgenic models of the cognate disorders and will be in one of the following areas: (i) Development of AAV micro-dystrophin gene vectors for DMD gene therapy; (ii) Inhibition of myostatin (GDF8) for treatment of muscle wasting diseases; (iii) Development of antisense therapeutics to modulate RNA splicing; (iv) Development of DNA and adenovirus vector vaccines for HIV/AIDS.

If you are interested, please visit my web pages for furhter information http://pure.rhul.ac.uk/portal/en/persons/george-dickson_f8b3f42b-1968-4521-8c46-f9250bd7fc63.html

Funding Notes:


Refer to College website http://www.rhul.ac.uk/studyhere/researchdegrees/feesandfunding/home.aspx

References:


Koo T, Okada T, Athanasopoulos T, Foster H, Takeda S, Dickson G. Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog. J Gene Medicine (in Press).
2. Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet. 2011 Aug 13;378(9791):595-605. Epub 2011 Jul 23. PubMed PMID: 21784508; PubMed Central PMCID: PMC3156980.
3. Malerba A, Boldrin L, Dickson G. Long-term systemic administration of unconjugated morpholino oligomers for therapeutic expression of dystrophin by exon skipping in skeletal muscle: implications for cardiac muscle integrity. Nucleic Acid Ther. 2011 Aug;21(4):293-8. PubMed PMID: 21851223.
4. Koo T, Malerba A, Athanasopoulos T, Trollet C, Boldrin L, Ferry A, Popplewell L, Foster H, Foster K, Dickson G. Delivery of AAV2/9-Microdystrophin Genes Incorporating Helix 1 of the Coiled-Coil Motif in the C-Terminal Domain of Dystrophin Improves Muscle Pathology and Restores the Level of α1-Syntrophin and α-Dystrobrevin in Skeletal Muscles of mdx Mice. Hum Gene Ther. 2011 May 25. [Epub ahead of print] PubMed PMID: 21453126..
5. Malerba A, Sharp PS, Graham IR, Arechavala-Gomeza V, Foster K, Muntoni F, Wells DJ, Dickson G. Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx Mice. Mol Ther. 2011 Feb;19(2):345-54. Epub 2010 Nov 23. PubMed PMID: 21102560.
6. Kang JK, Malerba A, Popplewell L, Foster K, Dickson G. Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. Mol Ther. 2011 Jan;19(1):159-64. Epub 2010 Oct 5. PubMed PMID: 20924365.

Research Assessment Exercise (RAE) 2008 Results


Unit of Assessment: Biological Sciences What is the RAE?
FTE Category A Staff Submitted 4* 3* 2* 1* U/C
22 15% 55% 25% 5% 0%


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