Global Utilities

La Trobe University
Science, Technology and Engineering

Honours Projects 2010

Dr Belinda Abbott


Synthesis of Peptide Nucleic Acid Conjugates

(In collaboration with Prof. S. Langford, Monash University)

Antisense therapy involves the design of a complementary molecule for binding to a specific DNA or mRNA sequence, allowing the production of a rogue protein being coded by a disease-causing gene to be “switched off” (see diagram).

diagram
Peptide nucleic acids (PNA) possess a neutral 2-amino ethylglycine back-bone and have higher affinity, stability and specificity than other molecules which have been investigated for this purpose.

Amyotrophic lateral sclerosis (ALS), the common form of motor neurone disease, is a progressive and fatal paralytic disorder. Patients with ALS have increasing problems with moving, swallowing, speaking and breathing.

Antisense PNA for the mRNA target SOD1G93A, found in some ALS patients, has already been demonstrated as having neuroprotective properties both in vitro and in vivo (Cheah 2007). These promising early results have indicated that greater efficacy could be achieved if cellular uptake of PNA was improved. This project is interested at looking at novel methods of PNA delivery through the use of lipid soluble conjugates such as Vitamin E and K.