Literature DB >> 20679391

Antisense oligonucleotides and spinal muscular atrophy: skipping along.

Arthur H M Burghes1, Vicki L McGovern.   

Abstract

Antisense oligonucleotides (ASOs) can be used to alter the splicing of a gene and either restore production of a required protein or eliminate a toxic product. In this issue of Genes & Development, Hua and colleagues (pp. 1634-1644) show that ASOs directed against an intron splice silencer (ISS) in the survival motor neuron 2 (SMN2) gene alter the amount of full-length SMN transcript in the nervous system, restoring SMN to levels that could correct spinal muscular atrophy (SMA).

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Year:  2010        PMID： 20679391      PMCID： PMC2912553          DOI： 10.1101/gad.1961710

Source DB:  PubMed          Journal:  Genes Dev        ISSN： 0890-9369            Impact factor:   11.361


  45 in total

Review 1.  Gene-targeted therapies for the central nervous system.

Authors:  Timothy M Miller; Richard A Smith; Holly Kordasiewicz; Brian K Kaspar
Journal:  Arch Neurol       Date:  2008-02-11

2.  A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.

Authors:  U R Monani; C L Lorson; D W Parsons; T W Prior; E J Androphy; A H Burghes; J D McPherson
Journal:  Hum Mol Genet       Date:  1999-07       Impact factor: 6.150

3.  SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN.

Authors:  Thanh T Le; Lan T Pham; Matthew E R Butchbach; Honglai L Zhang; Umrao R Monani; Daniel D Coovert; Tatiana O Gavrilina; Lei Xing; Gary J Bassell; Arthur H M Burghes
Journal:  Hum Mol Genet       Date:  2005-02-09       Impact factor: 6.150

4.  Local dystrophin restoration with antisense oligonucleotide PRO051.

Authors:  Judith C van Deutekom; Anneke A Janson; Ieke B Ginjaar; Wendy S Frankhuizen; Annemieke Aartsma-Rus; Mattie Bremmer-Bout; Johan T den Dunnen; Klaas Koop; Anneke J van der Kooi; Nathalie M Goemans; Sjef J de Kimpe; Peter F Ekhart; Edna H Venneker; Gerard J Platenburg; Jan J Verschuuren; Gert-Jan B van Ommen
Journal:  N Engl J Med       Date:  2007-12-27       Impact factor: 91.245

5.  Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.

Authors:  Kevin D Foust; Emily Nurre; Chrystal L Montgomery; Anna Hernandez; Curtis M Chan; Brian K Kaspar
Journal:  Nat Biotechnol       Date:  2008-12-21       Impact factor: 54.908

6.  Regulation of SMN protein stability.

Authors:  Barrington G Burnett; Eric Muñoz; Animesh Tandon; Deborah Y Kwon; Charlotte J Sumner; Kenneth H Fischbeck
Journal:  Mol Cell Biol       Date:  2008-12-22       Impact factor: 4.272

7.  Motor neuron disease: The curious ways of ALS.

Authors:  Magdalini Polymenidou; Don W Cleveland
Journal:  Nature       Date:  2008-07-17       Impact factor: 49.962

8.  Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

Authors:  Yimin Hua; Timothy A Vickers; Hazeem L Okunola; C Frank Bennett; Adrian R Krainer
Journal:  Am J Hum Genet       Date:  2008-03-27       Impact factor: 11.025

9.  Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation.

Authors:  Kathrin Meyer; Julien Marquis; Judith Trüb; Rachel Nlend Nlend; Sonia Verp; Marc-David Ruepp; Hans Imboden; Isabelle Barde; Didier Trono; Daniel Schümperli
Journal:  Hum Mol Genet       Date:  2008-11-13       Impact factor: 6.150

10.  Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs.

Authors:  Francesca Gabanella; Matthew E R Butchbach; Luciano Saieva; Claudia Carissimi; Arthur H M Burghes; Livio Pellizzoni
Journal:  PLoS One       Date:  2007-09-26       Impact factor: 3.240
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  15 in total

1.  Temporal requirement for high SMN expression in SMA mice.

Authors:  Thanh T Le; Vicki L McGovern; Isaac E Alwine; Xueyong Wang; Aurelie Massoni-Laporte; Mark M Rich; Arthur H M Burghes
Journal:  Hum Mol Genet       Date:  2011-06-13       Impact factor: 6.150

2.  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

3.  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

4.  Alternative splicing in spinal muscular atrophy underscores the role of an intron definition model.

Authors:  Natalia N Singh; Ravindra N Singh
Journal:  RNA Biol       Date:  2011-07-01       Impact factor: 4.652

Review 5.  New therapeutic approaches to spinal muscular atrophy.

Authors:  Aga Lewelt; Tara M Newcomb; Kathryn J Swoboda
Journal:  Curr Neurol Neurosci Rep       Date:  2012-02       Impact factor: 5.081

6.  Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery.

Authors:  Jacqueline J Glascock; Erkan Y Osman; Mary J Wetz; Megan M Krogman; Monir Shababi; Christian L Lorson
Journal:  Hum Gene Ther       Date:  2012-01-26       Impact factor: 5.695

7.  SMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.

Authors:  John T Kissel; Bakri Elsheikh; Wendy M King; Miriam Freimer; Charles B Scott; Stephen J Kolb; Sandra P Reyna; Thomas O Crawford; Louise R Simard; Kristin J Krosschell; Gyula Acsadi; Mary K Schroth; Guy D'Anjou; Bernard LaSalle; Thomas W Prior; Susan Sorenson; Jo Anne Maczulski; Kathryn J Swoboda
Journal:  Muscle Nerve       Date:  2014-02       Impact factor: 3.217

8.  Chronic Treatment with the AMPK Agonist AICAR Prevents Skeletal Muscle Pathology but Fails to Improve Clinical Outcome in a Mouse Model of Severe Spinal Muscular Atrophy.

Authors:  Clàudia Cerveró; Neus Montull; Olga Tarabal; Lídia Piedrafita; Josep E Esquerda; Jordi Calderó
Journal:  Neurotherapeutics       Date:  2016-01       Impact factor: 7.620

Review 9.  Targeting RNA to treat neuromuscular disease.

Authors:  Francesco Muntoni; Matthew J A Wood
Journal:  Nat Rev Drug Discov       Date:  2011-08-01       Impact factor: 84.694

10.  Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain.

Authors:  Ioannis Zalachoras; Gwendolynn Grootaers; Lisa T C M van Weert; Yves Aubert; Suzanne R de Kreij; Nicole A Datson; Willeke M C van Roon-Mom; Annemieke Aartsma-Rus; Onno C Meijer
Journal:  BMC Neurosci       Date:  2013-01-07       Impact factor: 3.288
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