Literature DB >> 19330716

Splicing therapeutics in SMN2 and APOB.

Bernard Khoo1, Adrian R Krainer.   

Abstract

Splicing therapeutics are defined as the deliberate modification of RNA splicing to achieve therapeutic goals. Various techniques for splicing therapeutics have been described, and most of these involve the use of antisense oligonucleotide-based compounds that target key elements in the pre-mRNA to control splicing in the nucleus. In this review, recent developments in splicing therapeutics for the treatment of two specific diseases are described: correcting the alternative splicing of survival of motor neuron (SMN)2 pre-mRNA to compensate for the defective SMN1 gene in spinal muscular atrophy, and re-engineering the splicing of apolipoprotein B pre-mRNA to lower circulating cholesterol levels.

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Year:  2009        PMID： 19330716      PMCID： PMC3140428     

Source DB:  PubMed          Journal:  Curr Opin Mol Ther        ISSN： 1464-8431


  63 in total

1.  Antisense properties of peptide nucleic acid.

Authors:  P E Nielsen
Journal:  Methods Enzymol       Date:  2000       Impact factor: 1.600

Review 2.  Pre-mRNA splicing in the new millennium.

Authors:  M L Hastings; A R Krainer
Journal:  Curr Opin Cell Biol       Date:  2001-06       Impact factor: 8.382

Review 3.  Sorting out the complexity of SR protein functions.

Authors:  B R Graveley
Journal:  RNA       Date:  2000-09       Impact factor: 4.942

4.  Htra2-beta 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2).

Authors:  Y Hofmann; C L Lorson; S Stamm; E J Androphy; B Wirth
Journal:  Proc Natl Acad Sci U S A       Date:  2000-08-15       Impact factor: 11.205

Review 5.  Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders.

Authors:  Ronald J Mandel; Fredric P Manfredsson; Kevin D Foust; Aaron Rising; Sharon Reimsnider; Kevin Nash; Corinna Burger
Journal:  Mol Ther       Date:  2006-01-18       Impact factor: 11.454

6.  Antisense oligonucleotide therapy for neurodegenerative disease.

Authors:  Richard A Smith; Timothy M Miller; Koji Yamanaka; Brett P Monia; Thomas P Condon; Gene Hung; Christian S Lobsiger; Chris M Ward; Melissa McAlonis-Downes; Hongbing Wei; Ed V Wancewicz; C Frank Bennett; Don W Cleveland
Journal:  J Clin Invest       Date:  2006-07-27       Impact factor: 14.808

7.  Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice.

Authors:  Jennifer Roberts; Enzo Palma; Peter Sazani; Henrik Ørum; Moo Cho; Ryszard Kole
Journal:  Mol Ther       Date:  2006-07-18       Impact factor: 11.454

Review 8.  An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA).

Authors:  B Wirth
Journal:  Hum Mutat       Date:  2000       Impact factor: 4.878

Review 9.  RNA editing: cytidine to uridine conversion in apolipoprotein B mRNA.

Authors:  A Chester; J Scott; S Anant; N Navaratnam
Journal:  Biochim Biophys Acta       Date:  2000-11-15

10.  Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron.

Authors:  Nirmal K Singh; Natalia N Singh; Elliot J Androphy; Ravindra N Singh
Journal:  Mol Cell Biol       Date:  2006-02       Impact factor: 4.272
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  16 in total

Review 1.  Developments in RNA splicing and disease.

Authors:  Michael G Poulos; Ranjan Batra; Konstantinos Charizanis; Maurice S Swanson
Journal:  Cold Spring Harb Perspect Biol       Date:  2011-01-01       Impact factor: 10.005

Review 2.  Impairment of pre-mRNA splicing in liver disease: mechanisms and consequences.

Authors:  Carmen Berasain; Saioa Goñi; Josefa Castillo; María Ujue Latasa; Jesús Prieto; Matías A Avila
Journal:  World J Gastroenterol       Date:  2010-07-07       Impact factor: 5.742

3.  Mechanisms of Neuronal Alternative Splicing and Strategies for Therapeutic Interventions.

Authors:  Eduardo Javier Lopez Soto; Michael J Gandal; Thomas Gonatopoulos-Pournatzis; Elizabeth A Heller; Diou Luo; Sika Zheng
Journal:  J Neurosci       Date:  2019-10-16       Impact factor: 6.167

Review 4.  Spinal muscular atrophy: mechanisms and therapeutic strategies.

Authors:  Christian L Lorson; Hansjorg Rindt; Monir Shababi
Journal:  Hum Mol Genet       Date:  2010-04-13       Impact factor: 6.150

5.  Therapy development in spinal muscular atrophy.

Authors:  Michael Sendtner
Journal:  Nat Neurosci       Date:  2010-07       Impact factor: 24.884

6.  The little elongation complex regulates small nuclear RNA transcription.

Authors:  Edwin R Smith; Chengqi Lin; Alexander S Garrett; Janet Thornton; Nima Mohaghegh; Deqing Hu; Jessica Jackson; Anita Saraf; Selene K Swanson; Christopher Seidel; Laurence Florens; Michael P Washburn; Joel C Eissenberg; Ali Shilatifard
Journal:  Mol Cell       Date:  2011-12-23       Impact factor: 17.970

7.  Integrative genome-wide analysis of the determinants of RNA splicing in kidney renal clear cell carcinoma.

Authors:  Kjong-Van Lehmann; André Kahles; Cyriac Kandoth; William Lee; Nikolaus Schultz; Oliver Stegle; Gunnar Rätsch
Journal:  Pac Symp Biocomput       Date:  2015

Review 8.  Therapeutic RNA manipulation in liver disease.

Authors:  Thomas A Kerr; Nicholas O Davidson
Journal:  Hepatology       Date:  2010-03       Impact factor: 17.425

9.  Regulation of the mutually exclusive exons 8a and 8 in the CaV1.2 calcium channel transcript by polypyrimidine tract-binding protein.

Authors:  Zhen Zhi Tang; Shalini Sharma; Sika Zheng; Geetanjali Chawla; Julia Nikolic; Douglas L Black
Journal:  J Biol Chem       Date:  2011-01-31       Impact factor: 5.157

Review 10.  Development of therapeutic splice-switching oligonucleotides.

Authors:  Petra Disterer; Adrianna Kryczka; Yuqi Liu; Yusef E Badi; Jessie J Wong; James S Owen; Bernard Khoo
Journal:  Hum Gene Ther       Date:  2014-06-19       Impact factor: 5.695
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