Yin, H., Saleh, A.F., Betts, C., Camiletti, P., Seow, Y., Ashraf, A., Arzumanov, A., Gait, M.J., and Wood, M.J.A. (2011)
Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.
Molecular Therapy, 19, doi:10.1038/mt.2011.79.
Torres, A.G., Fabani, M.M., Vigorito, E. and Gait, M.J. (2011)
MicroRNA fate upon targeting with anti-miRNA oligonuceotides as revealed by an improved Northern-blot based method for miRNA detection.
RNA 17, 933-943.
Fabani, M.M., Abreu-Goodger, C., Williams, D., Lyons, P.A., Torres, A., Smith, K.G.C., Enright, A., Gait, M.J., and Vigorito, E. (2010)
Efficient inhibition of miR-155 with peptide nucleic acids in primary B-cells and in vivo.
Nucleic Acids Res. 38, 4466-4475.
Visit my group page here
Contact me by email:
This e-mail address is being protected from spambots. You need JavaScript enabled to view it
Group Members
- Andrey Arzoumanov
- Amer Saleh
- Peter Deuss
- Thibault Coursindel
- Peter Jarver
|
|
|
Our work focuses on the chemical synthesis of modified oligonucleotides, siRNA, peptide nucleic acids (PNA), and their peptide conjugates to target essential RNA structures inside cells for a variety of potential therapeutic applications.
Two studies are currently in progress. The first study concerns steric block antisense targeting by PNA conjugated to cell-penetrating peptides to mediate alternative RNA splicing in the cell nucleus. One application is exon skipping of the dystrophin gene in a mouse model of Duchenne muscular mystrophy, a genetic disease manifest in young boys for which there is currently no cure.
The second study is the synthesis of PNA and other analogues as steric block agents targeted to microRNA, important newly discovered regulators of gene expression, with applications for example in liver disease, cancer and neuromuscular diseases.
|
|
Synthetic oligonucleotide analogues can be exploited to block microRNA activity in cells. Cell-penetrating peptide-PNA conjugates may be directed to the cell nucleus to inhibit microRNA transcription or to block precursor maturation, or PNA with attached Lys residues delivered to the cytosol to block microRNA loading into miRISC or mRNA target recognition/binding.
|
|
Group Leaders 
