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Morpholino-driven gene editing: a new horizon for disease treatment and prevention
Journal article   Peer reviewed

Morpholino-driven gene editing: a new horizon for disease treatment and prevention

Ekaterina Subbotina, Siva Rama Krishna Koganti, Denice Hodgson-Zingman and Leonid V Zingman
Clinical pharmacology and therapeutics, Vol.99(1), pp.21-25
01/2016
DOI: 10.1002/cpt.276
PMCID: PMC4720544
PMID: 26474085

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Abstract

The development of genetic and molecular biology tools permitting the connection of specific genes to their functions has accelerated our understanding of molecular pathways underlying health and disease. The resulting gains in knowledge have propelled gene targeting to the forefront of promising therapeutic strategies. Here we discuss the uniquely powerful and adaptable approach of morpholino-driven modification of normal and mutant gene expression as a pathway to health.
gene silencing skeletal muscle vivo-morpholino oligonucleotide ATP-sensitive potassium channel

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