FNAPerth2018

International Symposium on Functional Nucleic Acids: From Laboratory to Targeted Molecular Therapy, 22nd & 23rd November 2018, Perth, Australia.

[FNAPerth2018 @FnaPerth2015]

Our focus is on nucleic acid chemistry & biology incl. novel modified nucleotides, therapeutic & diagnostic appl. of nucleic acid constructs

[FNAPerth2018 @FnaPerth2015]

See you in 2021!

[FNAPerth2018 @FnaPerth2015]

Closing remarks from Steve Arnott, CEO of Perron Institute. 'On the way to wonderful, you'll pass 'all right'. Stop and take a look around' quote from Bill Withers

[FNAPerth2018 @FnaPerth2015]

Special thanks to the team behind the conference

[FNAPerth2018 @FnaPerth2015]

Joanna Macdonald, Naoke Abe, Justin Henri all received 3rd prize, Jessica Cale came 2nd and 1st prize went to Bao Le

[FNAPerth2018 @FnaPerth2015]

There are 5 prizes for posters and @rakeshveedu is giving out poster prizes, sponsored by Molecules - a MDPI journal

[FNAPerth2018 @FnaPerth2015]

And that was the end of the scientific program. Time for awards and thank you's.

[FNAPerth2018 @FnaPerth2015]

Wilton: large number of studies currently underway in the lab

[FNAPerth2018 @FnaPerth2015]

Wilton: loss of exon identity is a common mechanism of human inherited disease. Currently looking at Marfan's disease, Ehler-Danlos syndrome, ALS, MS....

[FNAPerth2018 @FnaPerth2015]

Wilton: it's not a cure but can turn DMD into Becker. Gives stabilisation of respiratory function < it's great seeing the smiling faces of boys doing things they couldn't even 9 weeks previously

[FNAPerth2018 @FnaPerth2015]

Wilton: Eteplirsen is a morpholino oligomer excises the human dystrophin exon 51 from the mature mRNA fast tracked by FDA. Some boys have been treated for 9 years and are still ambulatory

[FNAPerth2018 @FnaPerth2015]

Wilton: gene deletions that maintain the dystrophin reading frame generally result in milder Becker MD and it may be possible to develop antisense oligos to specifically redirect dystrophin pre-mRNA processing to restore reading frame

[FNAPerth2018 @FnaPerth2015]

Wilton: in Becker you have a partially functional protein

[FNAPerth2018 @FnaPerth2015]

Wilton: Becker muscular dystrophy is a milder form of DMD

[FNAPerth2018 @FnaPerth2015]

Last up is Steve Wilton who is providing an update on Duchenne muscular dystrophy exon skipping

[FNAPerth2018 @FnaPerth2015]

Shigdar: as final proof, attached doxorubicin to the aptamer and confirmed internalisation in only cancer cells in the brain within 60 minutes of a tail vein injection in a breast cancer brain metastases model

[FNAPerth2018 @FnaPerth2015]

Shigdar: tested this bifunctional aptamer in both in vitro and in vivo and confirmed functionality

[FNAPerth2018 @FnaPerth2015]

Shigdar: this is important for treating brain metastases so as to protect healthy brain cells

[FNAPerth2018 @FnaPerth2015]

Shigdar: took a transferrin receptor aptamer, truncated it to smallest functional unit and joined it to our EpCAM to generate a bifunctional aptamer

[FNAPerth2018 @FnaPerth2015]

Shigdar: there are a number of ways being investigated to get into the brain - we are using a non-invasive receptor mediated transcytosis approach

[FNAPerth2018 @FnaPerth2015]

Shigdar: but the problem is that more patients are being diagnosed with brain metastases and very few treatments can get into the brain