Literature DB >> 24171481

A chemical view of oligonucleotides for exon skipping and related drug applications.

Peter Järver1, Liz O'Donovan, Michael J Gait.   

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Year:  2013        PMID： 24171481      PMCID： PMC3923385          DOI： 10.1089/nat.2013.0454

Source DB:  PubMed          Journal:  Nucleic Acid Ther        ISSN： 2159-3337            Impact factor:   5.486


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  85 in total

1.  Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Authors:  Yimin Hua; Kentaro Sahashi; Gene Hung; Frank Rigo; Marco A Passini; C Frank Bennett; Adrian R Krainer
Journal:  Genes Dev       Date:  2010-07-12       Impact factor: 11.361

2.  Preparation and properties of 2',5'-linked oligonucleotide analogues containing 3'-O,4'-C-methyleneribonucleosides.

Authors:  S Obika; K Morio; Y Hari; T Imanishi
Journal:  Bioorg Med Chem Lett       Date:  1999-02-22       Impact factor: 2.823

3.  Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy.

Authors:  Erkan Y Osman; Pei-Fen Yen; Christian L Lorson
Journal:  Mol Ther       Date:  2011-10-25       Impact factor: 11.454

4.  A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.

Authors:  Paul N Porensky; Chalermchai Mitrpant; Vicki L McGovern; Adam K Bevan; Kevin D Foust; Brain K Kaspar; Stephen D Wilton; Arthur H M Burghes
Journal:  Hum Mol Genet       Date:  2011-12-20       Impact factor: 6.150

5.  Polyethylenimine-modified pluronics (PCMs) improve morpholino oligomer delivery in cell culture and dystrophic mdx mice.

Authors:  Mingxing Wang; Bo Wu; Peijuan Lu; Caryn Cloer; Jay D Tucker; Qilong Lu
Journal:  Mol Ther       Date:  2012-11-20       Impact factor: 11.454

6.  Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA.

Authors:  Thurman M Wheeler; Krzysztof Sobczak; John D Lueck; Robert J Osborne; Xiaoyan Lin; Robert T Dirksen; Charles A Thornton
Journal:  Science       Date:  2009-07-17       Impact factor: 47.728

7.  Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse.

Authors:  HaiFang Yin; Corinne Betts; Amer F Saleh; Gabriela D Ivanova; Hyunil Lee; Yiqi Seow; Dalsoo Kim; Michael J Gait; Matthew J A Wood
Journal:  Mol Ther       Date:  2010-01-12       Impact factor: 11.454

8.  Development of novel bioanalytical methods to determine the effective concentrations of phosphorodiamidate morpholino oligomers in tissues and cells.

Authors:  Frederick J Schnell; Stacy L Crumley; Dan V Mourich; Patrick L Iversen
Journal:  Biores Open Access       Date:  2013-02

9.  Improved Performance of Anti-miRNA Oligonucleotides Using a Novel Non-Nucleotide Modifier.

Authors:  Kim A Lennox; Richard Owczarzy; Derek M Thomas; Joseph A Walder; Mark A Behlke
Journal:  Mol Ther Nucleic Acids       Date:  2013-08-27       Impact factor: 10.183

10.  Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates.

Authors:  Ellen Marie Straarup; Niels Fisker; Maj Hedtjärn; Marie W Lindholm; Christoph Rosenbohm; Vibeke Aarup; Henrik Frydenlund Hansen; Henrik Ørum; Jens B Rode Hansen; Troels Koch
Journal:  Nucleic Acids Res       Date:  2010-07-08       Impact factor: 16.971
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  35 in total

1.  Modulation of Splicing by Single-Stranded Silencing RNAs.

Authors:  Jing Liu; Jiaxin Hu; Jessica A Hicks; Thazha P Prakash; David R Corey
Journal:  Nucleic Acid Ther       Date:  2015-03-10       Impact factor: 5.486

2.  Synthesis, Characterization, and Function of an RNA-Based Transfection Reagent.

Authors:  Harsh V Jain; Jessica F Boehler; Kanneboyina Nagaraju; Serge L Beaucage
Journal:  Curr Protoc Nucleic Acid Chem       Date:  2018-03

3.  Duchenne muscular dystrophy drugs at the crossroads, as newer agents advance.

Authors:  Cormac Sheridan
Journal:  Nat Biotechnol       Date:  2016-07-12       Impact factor: 54.908

Review 4.  Using Genome Sequence to Enable the Design of Medicines and Chemical Probes.

Authors:  Alicia J Angelbello; Jonathan L Chen; Jessica L Childs-Disney; Peiyuan Zhang; Zi-Fu Wang; Matthew D Disney
Journal:  Chem Rev       Date:  2018-01-11       Impact factor: 60.622

Review 5.  The delivery of therapeutic oligonucleotides.

Authors:  Rudolph L Juliano
Journal:  Nucleic Acids Res       Date:  2016-04-15       Impact factor: 16.971

Review 6.  Non-coding RNAs as drug targets.

Authors:  Masayuki Matsui; David R Corey
Journal:  Nat Rev Drug Discov       Date:  2016-07-22       Impact factor: 84.694

7.  Welcome to the splice age: antisense oligonucleotide-mediated exon skipping gains wider applicability.

Authors:  Elizabeth M McNally; Eugene J Wyatt
Journal:  J Clin Invest       Date:  2016-03-21       Impact factor: 14.808

8.  An amphipathic trans-acting phosphorothioate RNA element delivers an uncharged phosphorodiamidate morpholino sequence in mdx mouse myotubes.

Authors:  H V Jain; J F Boehler; D Verthelyi; K Nagaraju; S L Beaucage
Journal:  RSC Adv       Date:  2017-09-04       Impact factor: 3.361

9.  An Amphipathic trans-Acting Phosphorothioate DNA Element Delivers Uncharged PNA and PMO Nucleic Acid Sequences in Mammalian Cells.

Authors:  Harsh V Jain; Serge L Beaucage
Journal:  Curr Protoc Nucleic Acid Chem       Date:  2016-03

Review 10.  Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy.

Authors:  Maria Sofia Falzarano; Chiara Passarelli; Alessandra Ferlini
Journal:  Nucleic Acid Ther       Date:  2014-02       Impact factor: 5.486
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