Antisense-mediated exon skipping: Networking to meet opportunities and to overcome challenges

Nucleic Acid Therapeutics

Volumn 24, Issue 1, 2014, Pages 1-3

  Aartsma Rus, Annemieke ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ISOPROTEIN;

ANTISENSE THERAPY; DUCHENNE MUSCULAR DYSTROPHY; EDITORIAL; EXON SKIPPING; GENE MUTATION; HUMAN; HUNTINGTON CHOREA; NEUROLOGIC DISEASE; NEUROMUSCULAR DISEASE; PERSONALIZED MEDICINE; PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEIN ANALYSIS; REGISTER; SPINAL MUSCULAR ATROPHY; THERAPEUTIC MISCONCEPTION;

COOPERATIVE BEHAVIOR; EXONS; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; RARE DISEASES; TARGETED GENE REPAIR;

EID: 84893536025     PISSN: 21593337     EISSN: None     Source Type: Journal    
DOI: 10.1089/nat.2014.1500     Document Type: Editorial

References (10)

1. BESTAS, B., MCCLOREY, G., TEDEBARK, U., MORENO, P.M.D., ROBERTS, T.C., HAMMOND, S.M., SMITH, C.I.E., WOOD, M.J.A., and EL ANDALOUSSI, S. (2013). Design and application of bispecific splice switching oligonucleotides. Nucleic Acid Ther. 24, 13-24.
2. EVERS, M.M., TRAN, H.D., ZALACHORAS, I., MEIJER, O.C., DEN DUNNEN, J.T., VAN OMMEN, G.J., AARTSMA-RUS, A., and VAN ROON-MOM, W.M. (2013). Preventing formation of toxic n-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification. Nucleic Acid Ther. 24, 4-12.
3. FALZARANO, M.S., PASSARELLI, C., and FERLINI, A. (2014). Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy. Nucleic Acid Ther. 24, 87-100.
4. JARVER, P., O'DONOVAN, L., and GAIT, M.J. (2013). A chemical view of oligonucleotides for exon skipping and related drug applications. Nucleic Acid Ther. 24, 37-47.
5. JIRKA, S.M., HEEMSKERK, H., TANGANYIKA-DE WINTER, C.L., MUILWIJK, D., PANG, K.H., DE VISSER, P.C., JANSON, A., KARNAOUKH, T.G., VERMUE, R., 'T HOEN, P.A. et al. (2013). Peptide conjugation of 2'-Omethyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice. Nucleic Acid Ther. 24, 25-36.
6. KOO, T., and WOOD, M.J. (2013). Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy. Hum. Gene Ther. 24, 479-488.
7. MUNTONI, F. (2010). The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009. Neuromuscul. Disord. 20, 355-362.
8. SIVA, K., COVELLO, G., and DENTI, M.A. (2014). Exonskipping antisense oligonucleotides to correct missplicing in neurogenetic diseases. Nucleic Acid Ther. 24, 69-86.
9. SPITALI, P., and AARTSMA-RUS, A. (2012). Splice modulating therapies for human disease. Cell 148, 1085-1088.
10. VAN ROON-MOM, W.M., and AARTSMA-RUS, A. (2012). Overview on applications of antisense-mediated exon skipping. Methods Mol. Biol. 867, 79-96.