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Items: 14

1.

Delivery is key: lessons learnt from developing splice-switching antisense therapies.

Godfrey C, Desviat LR, Smedsrød B, Piétri-Rouxel F, Denti MA, Disterer P, Lorain S, Nogales-Gadea G, Sardone V, Anwar R, El Andaloussi S, Lehto T, Khoo B, Brolin C, van Roon-Mom WM, Goyenvalle A, Aartsma-Rus A, Arechavala-Gomeza V.

EMBO Mol Med. 2017 May;9(5):545-557. doi: 10.15252/emmm.201607199. Review.

2.

Dieter C. Gruenert, PhD (1949-2016).

Disterer P, Kay MA, Parker GC.

Nucleic Acid Ther. 2016 Aug;26(4):266-7. doi: 10.1089/nat.2016.29002.par. No abstract available.

PMID:
27500925
3.

Distinct patterns of heparan sulphate in pancreatic islets suggest novel roles in paracrine islet regulation.

Theodoraki A, Hu Y, Poopalasundaram S, Oosterhof A, Guimond SE, Disterer P, Khoo B, Hauge-Evans AC, Jones PM, Turnbull JE, van Kuppevelt TH, Bouloux PM.

Mol Cell Endocrinol. 2015 Jan 5;399:296-310. doi: 10.1016/j.mce.2014.09.011. Epub 2014 Sep 16.

PMID:
25224485
4.

Development of therapeutic splice-switching oligonucleotides.

Disterer P, Kryczka A, Liu Y, Badi YE, Wong JJ, Owen JS, Khoo B.

Hum Gene Ther. 2014 Jul;25(7):587-98. doi: 10.1089/hum.2013.234. Epub 2014 Jun 19. Review.

5.

Pathogenetic mechanisms of amyloid A amyloidosis.

Simons JP, Al-Shawi R, Ellmerich S, Speck I, Aslam S, Hutchinson WL, Mangione PP, Disterer P, Gilbertson JA, Hunt T, Millar DJ, Minogue S, Bodin K, Pepys MB, Hawkins PN.

Proc Natl Acad Sci U S A. 2013 Oct 1;110(40):16115-20. doi: 10.1073/pnas.1306621110. Epub 2013 Aug 19.

6.

Exon skipping of hepatic APOB pre-mRNA with splice-switching oligonucleotides reduces LDL cholesterol in vivo.

Disterer P, Al-Shawi R, Ellmerich S, Waddington SN, Owen JS, Simons JP, Khoo B.

Mol Ther. 2013 Mar;21(3):602-9. doi: 10.1038/mt.2012.264. Epub 2013 Jan 15.

7.

Antisense-mediated exon-skipping to induce gene knockdown.

Disterer P, Khoo B.

Methods Mol Biol. 2012;867:289-305. doi: 10.1007/978-1-61779-767-5_19.

PMID:
22454069
8.

Oligonucleotide-mediated gene editing is underestimated in cells expressing mutated green fluorescent protein and is positively associated with target protein expression.

Disterer P, Papaioannou I, Evans VC, Simons JP, Owen JS.

J Gene Med. 2012 Feb;14(2):109-19. doi: 10.1002/jgm.1639.

PMID:
22228477
9.

Validation of oligonucleotide-mediated gene editing.

Disterer P, Simons JP, Owen JS.

Gene Ther. 2009 Jun;16(6):824-6. doi: 10.1038/gt.2009.32. Epub 2009 Apr 2.

PMID:
19340016
11.

The presence of valine at residue 129 in human prion protein accelerates amyloid formation.

Baskakov I, Disterer P, Breydo L, Shaw M, Gill A, James W, Tahiri-Alaoui A.

FEBS Lett. 2005 May 9;579(12):2589-96. Epub 2005 Apr 8.

12.
13.

Multigene RNA vector based on coronavirus transcription.

Thiel V, Karl N, Schelle B, Disterer P, Klagge I, Siddell SG.

J Virol. 2003 Sep;77(18):9790-8.

14.

Characterization of 2'-fluoro-RNA aptamers that bind preferentially to disease-associated conformations of prion protein and inhibit conversion.

Rhie A, Kirby L, Sayer N, Wellesley R, Disterer P, Sylvester I, Gill A, Hope J, James W, Tahiri-Alaoui A.

J Biol Chem. 2003 Oct 10;278(41):39697-705. Epub 2003 Aug 5.

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