Opportunities and challenges for antisense oligonucleotide therapies

Liliana Matos, Vânia Gonçalves, Eugénia Pinto, Francisco Laranjeira, Maria João Prata, Peter Jordan, Lourdes R. Desviat, Belén Pérez, Sandra Alves, Data in support of a functional analysis of splicing mutations in the IDS gene and the use of antisense oligonucleotides to exploit an alternative therapy for MPS II Data in Brief. ,vol. 5, pp. 810- 817 ,(2015) , 10.1016/J.DIB.2015.10.011

Liliana Matos, Vânia Gonçalves, Eugénia Pinto, Francisco Laranjeira, Maria João Prata, Peter Jordan, Lourdes R. Desviat, Belén Pérez, Sandra Alves, Functional analysis of splicing mutations in the IDS gene and the use of antisense oligonucleotides to exploit an alternative therapy for MPS II. Biochimica et Biophysica Acta. ,vol. 1852, pp. 2712- 2721 ,(2015) , 10.1016/J.BBADIS.2015.09.011

Frederick J Raal, Raul D Santos, Dirk J Blom, A David Marais, Min-Ji Charng, William C Cromwell, Robin H Lachmann, Daniel Gaudet, Ju L Tan, Scott Chasan-Taber, Diane L Tribble, JoAnn D Flaim, Stanley T Crooke, Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. The Lancet. ,vol. 375, pp. 998- 1006 ,(2010) , 10.1016/S0140-6736(10)60284-X

Anna Diaz-Font, Amparo Chabás, Daniel Grinberg, Lluïsa Vilageliu, RNAi-mediated inhibition of the glucosylceramide synthase (GCS) gene: A preliminary study towards a therapeutic strategy for Gaucher disease and other glycosphingolipid storage diseases. Blood Cells Molecules and Diseases. ,vol. 37, pp. 197- 203 ,(2006) , 10.1016/J.BCMD.2006.07.002

Suzie Lefebvre, Lydie Bürglen, Sophie Reboullet, Olivier Clermont, Philippe Burlet, Louis Viollet, Bernard Benichou, Corinne Cruaud, Philippe Millasseau, Massimo Zeviani, Denis Le Paslier, Jean Frézal, Daniel Cohen, Jean Weissenbach, Arnold Munnich, Judith Melki, Identification and characterization of a spinal muscular atrophy-determining gene Cell. ,vol. 80, pp. 155- 165 ,(1995) , 10.1016/0092-8674(95)90460-3

Sandra Brasil, Hiu Man Viecelli, David Meili, Anahita Rassi, Lourdes R. Desviat, Belen Pérez, Magdalena Ugarte, Beat Thöny, Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency. Human Mutation. ,vol. 32, pp. 1019- 1027 ,(2011) , 10.1002/HUMU.21529

Francesco Vetrini, Roberta Tammaro, Sergio Bondanza, Enrico M. Surace, Alberto Auricchio, Michele De Luca, Andrea Ballabio, Valeria Marigo, Aberrant splicing in the ocular albinism type 1 gene (OA1/GPR143) is corrected in vitro by morpholino antisense oligonucleotides. Human Mutation. ,vol. 27, pp. 420- 426 ,(2006) , 10.1002/HUMU.20303

Kevin Fitzgerald, Maria Frank-Kamenetsky, Svetlana Shulga-Morskaya, Abigail Liebow, Brian R Bettencourt, Jessica E Sutherland, Renta M Hutabarat, Valerie A Clausen, Verena Karsten, Jeffrey Cehelsky, Saraswathy V Nochur, Victor Kotelianski, Jay Horton, Timothy Mant, Joseph Chiesa, James Ritter, Malathy Munisamy, Akshay K Vaishnaw, Jared A Gollob, Amy Simon, Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial The Lancet. ,vol. 383, pp. 60- 68 ,(2014) , 10.1016/S0140-6736(13)61914-5

Pietro Spitali, Annemieke Aartsma-Rus, Splice Modulating Therapies for Human Disease Cell. ,vol. 148, pp. 1085- 1088 ,(2012) , 10.1016/J.CELL.2012.02.014

Arthur M. Krieg, Mechanisms and applications of immune stimulatory CpG oligodeoxynucleotides. Biochimica et Biophysica Acta. ,vol. 1489, pp. 107- 116 ,(1999) , 10.1016/S0167-4781(99)00147-5