Gene therapy with modified U1 small nuclear RNA.
In: Expert Review of Endocrinology & Metabolism, Jg. 12 (2017-05-01), Heft 3, S. 171-175
academicJournal
Zugriff:
Introduction: More than 15% of all disease-causing mutations result in mRNA splicing defects. U1 snRNA binds to the 5ʹ splice site (5’ss) through base pairing. Mutation-adapted U1 snRNA (with compensatory U1 snRNA changes) and exon-specific U1 snRNA (complementary to intronic sequences) have been shown to suppress 5’ss mutations in cellular and animal models. Areas covered: The history, mechanism of action, and efficacy of U1 snRNA-mediated gene therapy are covered. The clinical utility of this technology and its limitations will be discussed. Expert commentary: Recently, gene therapies with mutation-adapted U1 snRNAs have been conducted on animal models, including aromaticl-amino acid decarboxylase deficiency and spinal muscular atrophy. However, although U1-mediated therapy has the advantage of maintaining the regulated expression of defective genes, its accuracy and efficacy needs to be improved before clinical application of this technique is possible. [ABSTRACT FROM AUTHOR]
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Gene therapy with modified U1 small nuclear RNA.
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Autor/in / Beteiligte Person: | Hwu, Wuh-Liang ; Lee, Yu-May ; Lee, Ni-Chung |
Zeitschrift: | Expert Review of Endocrinology & Metabolism, Jg. 12 (2017-05-01), Heft 3, S. 171-175 |
Veröffentlichung: | 2017 |
Medientyp: | academicJournal |
ISSN: | 1744-6651 (print) |
DOI: | 10.1080/17446651.2017.1316191 |
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