Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste

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...

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Published inJournal of investigative dermatology Vol. 132; no. 8; pp. 1959 - 1966
Main Authors Wally, Verena, Murauer, Eva M., Bauer, Johann W.
Format Journal Article
LanguageEnglish
Published New York, NY Elsevier Inc 01.08.2012
Nature Publishing Group
Elsevier Limited
Subjects
Alleles
Animals
Biological and medical sciences
Carcinoma, Squamous Cell - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Dermatology
Epidermolysis bullosa
Genetic Diseases, Inborn - genetics
Genetic Techniques
Genetic Therapy - methods
Genetic Therapy - trends
Humans
Medical sciences
Mice
Models, Genetic
Mutation
Neurodegenerative diseases
Phenotype
Reviews
RNA
RNA Splicing
Spliceosomes - genetics
Spliceosomes - metabolism
Therapeutic applications
Trans-Splicing
Splicing
Dermatology
Treatment
Online AccessGet full text
ISSN0022-202X
1523-1747
1523-1747
DOI10.1038/jid.2012.101

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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.
AbstractList 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.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.
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.
Author Bauer, Johann W.
Murauer, Eva M.
Wally, Verena
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Snippet Spliceosome-mediated RNA trans-splicing (SMaRT) is an RNA-based technology to reprogram genes for diagnostic and therapeutic purposes. For the correction of...
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StartPage 1959
SubjectTerms Alleles
Animals
Biological and medical sciences
Carcinoma, Squamous Cell - genetics
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Dermatology
Epidermolysis bullosa
Genetic Diseases, Inborn - genetics
Genetic Techniques
Genetic Therapy - methods
Genetic Therapy - trends
Humans
Medical sciences
Mice
Models, Genetic
Mutation
Neurodegenerative diseases
Phenotype
Reviews
RNA
RNA Splicing
Spliceosomes - genetics
Spliceosomes - metabolism
Therapeutic applications
Trans-Splicing
Title Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste
URI https://dx.doi.org/10.1038/jid.2012.101
https://www.ncbi.nlm.nih.gov/pubmed/22495179
https://www.proquest.com/docview/1024954961
https://www.proquest.com/docview/1026688930
https://www.proquest.com/docview/1028034545
Volume 132
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