Compositions and methods for modulating SMN gene family expression


Ontology type: sgo:Patent     


Patent Info

DATE

2018-08-28T00:00

AUTHORS

Arthur M. Krieg , Romesh Subramanian , James McSwiggen , Jeannie T. Lee

ABSTRACT

Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2. More... »

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