Literature DB >> 22495179

Spliceosome-mediated trans-splicing: the therapeutic cut and paste.

Verena Wally1, Eva M Murauer, Johann W Bauer.   

Abstract

Spliceosome-mediated RNA trans-splicing (SMaRT) is an RNA-based technology to reprogram genes for diagnostic and therapeutic purposes. For the correction of genetic diseases, SMaRT offers several advantages over traditional gene-replacement strategies. SMaRT protocols have recently been used for in vitro phenotypic correction of a variety of genetic disorders, ranging from epidermolysis bullosa to neurodegenerative diseases. In vivo studies are currently bringing trans-splicing RNA therapy toward clinical application. In this review, we summarize the progress made toward the medical use of SMaRT and provide an outlook on its upcoming applications.

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Year:  2012        PMID： 22495179     DOI： 10.1038/jid.2012.101

Source DB:  PubMed          Journal:  J Invest Dermatol        ISSN： 0022-202X            Impact factor:   8.551


  33 in total

Review 1.  MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction.

Authors:  Verena Behrens-Gawlik; Giulia Mearini; Christina Gedicke-Hornung; Pascale Richard; Lucie Carrier
Journal:  Pflugers Arch       Date:  2013-12-12       Impact factor: 3.657

2.  The design and optimization of RNA trans-splicing molecules for skin cancer therapy.

Authors:  Christina Gruber; Ulrich Koller; Eva M Murauer; Stefan Hainzl; Clemens Hüttner; Thomas Kocher; Andrew P South; Helmut Hintner; Johann W Bauer
Journal:  Mol Oncol       Date:  2013-08-19       Impact factor: 6.603

3.  Single molecule RNA sequencing uncovers trans-splicing and improves annotations in Anopheles stephensi.

Authors:  X Jiang; A B Hall; J K Biedler; Z Tu
Journal:  Insect Mol Biol       Date:  2017-02-09       Impact factor: 3.585

Review 4.  Exploiting differential RNA splicing patterns: a potential new group of therapeutic targets in cancer.

Authors:  Nidhi Jyotsana; Michael Heuser
Journal:  Expert Opin Ther Targets       Date:  2017-12-20       Impact factor: 6.902

Review 5.  Cardiac myosin-binding protein C (MYBPC3) in cardiac pathophysiology.

Authors:  Lucie Carrier; Giulia Mearini; Konstantina Stathopoulou; Friederike Cuello
Journal:  Gene       Date:  2015-09-08       Impact factor: 3.688

6.  Old gene, new phenotype: splice-altering variants in CEACAM16 cause recessive non-syndromic hearing impairment.

Authors:  Kevin T Booth; Kimia Kahrizi; Hela Azaiez; Richard Jh Smith; Hossein Najmabadi
Journal:  J Med Genet       Date:  2018-04-27       Impact factor: 6.318

Review 7.  Gene therapy strategies in the treatment of hypertrophic cardiomyopathy.

Authors:  Maksymilian Prondzynski; Giulia Mearini; Lucie Carrier
Journal:  Pflugers Arch       Date:  2018-07-03       Impact factor: 3.657

Review 8.  Targeting RNA splicing for disease therapy.

Authors:  Mallory A Havens; Dominik M Duelli; Michelle L Hastings
Journal:  Wiley Interdiscip Rev RNA       Date:  2013-03-19       Impact factor: 9.957

Review 9.  Targets for therapy in sarcomeric cardiomyopathies.

Authors:  Jil C Tardiff; Lucie Carrier; Donald M Bers; Corrado Poggesi; Cecilia Ferrantini; Raffaele Coppini; Lars S Maier; Houman Ashrafian; Sabine Huke; Jolanda van der Velden
Journal:  Cardiovasc Res       Date:  2015-01-29       Impact factor: 10.787

10.  Trans-splicing correction of tau isoform imbalance in a mouse model of tau mis-splicing.

Authors:  María Elena Avale; Teresa Rodríguez-Martín; Jean-Marc Gallo
Journal:  Hum Mol Genet       Date:  2013-03-03       Impact factor: 6.150
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