Literature DB >> 27479734

New Reasons to Pursue the Therapeutic Potential of Synthetic Nucleic Acids for Neurological Diseases.

Jill Sergesketter Butler1, Marek Napierala2.   

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Year:  2016        PMID： 27479734      PMCID： PMC5056836          DOI： 10.1001/jamaneurol.2016.2571

Source DB:  PubMed          Journal:  JAMA Neurol        ISSN： 2168-6149            Impact factor:   18.302


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  14 in total

Review 1.  Antisense oligonucleotides in therapy for neurodegenerative disorders.

Authors:  Melvin M Evers; Lodewijk J A Toonen; Willeke M C van Roon-Mom
Journal:  Adv Drug Deliv Rev       Date:  2015-03-20       Impact factor: 15.470

2.  DNA sequence-specific polyamides alleviate transcription inhibition associated with long GAA.TTC repeats in Friedreich's ataxia.

Authors:  Ryan Burnett; Christian Melander; James W Puckett; Leslie S Son; Robert D Wells; Peter B Dervan; Joel M Gottesfeld
Journal:  Proc Natl Acad Sci U S A       Date:  2006-07-20       Impact factor: 11.205

3.  Histone deacetylase inhibitors reverse gene silencing in Friedreich's ataxia.

Authors:  David Herman; Kai Jenssen; Ryan Burnett; Elisabetta Soragni; Susan L Perlman; Joel M Gottesfeld
Journal:  Nat Chem Biol       Date:  2006-08-20       Impact factor: 15.040

4.  Stabilization of expanded (CTG)•(CAG) repeats by antisense oligonucleotides.

Authors:  Masayuki Nakamori; Geneviève Gourdon; Charles A Thornton
Journal:  Mol Ther       Date:  2011-10-04       Impact factor: 11.454

5.  Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.

Authors:  Sebahattin Cirak; Virginia Arechavala-Gomeza; Michela Guglieri; Lucy Feng; Silvia Torelli; Karen Anthony; Stephen Abbs; Maria Elena Garralda; John Bourke; Dominic J Wells; George Dickson; Matthew J A Wood; Steve D Wilton; Volker Straub; Ryszard Kole; Stephen B Shrewsbury; Caroline Sewry; Jennifer E Morgan; Kate Bushby; Francesco Muntoni
Journal:  Lancet       Date:  2011-07-23       Impact factor: 79.321

6.  R loops stimulate genetic instability of CTG.CAG repeats.

Authors:  Yunfu Lin; Sharon Y R Dent; John H Wilson; Robert D Wells; Marek Napierala
Journal:  Proc Natl Acad Sci U S A       Date:  2009-12-22       Impact factor: 11.205

Review 7.  Antisense oligonucleotides: treating neurodegeneration at the level of RNA.

Authors:  Sarah L DeVos; Timothy M Miller
Journal:  Neurotherapeutics       Date:  2013-07       Impact factor: 7.620

8.  C9orf72 nucleotide repeat structures initiate molecular cascades of disease.

Authors:  Aaron R Haeusler; Christopher J Donnelly; Goran Periz; Eric A J Simko; Patrick G Shaw; Min-Sik Kim; Nicholas J Maragakis; Juan C Troncoso; Akhilesh Pandey; Rita Sattler; Jeffrey D Rothstein; Jiou Wang
Journal:  Nature       Date:  2014-03-05       Impact factor: 49.962

9.  Activating frataxin expression by repeat-targeted nucleic acids.

Authors:  Liande Li; Masayuki Matsui; David R Corey
Journal:  Nat Commun       Date:  2016-02-04       Impact factor: 14.919

10.  Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich's ataxia.

Authors:  Joseph F Nabhan; Kristy M Wood; Varada P Rao; Jeffrey Morin; Surya Bhamidipaty; Timothy P LaBranche; Renea L Gooch; Fazli Bozal; Christine E Bulawa; Braydon C Guild
Journal:  Sci Rep       Date:  2016-02-17       Impact factor: 4.379
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