MODIFICATION OF THE DYSTROPHIN GENE AND USES THEREOF
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United States of America Patent
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Issued Date -
Sep 20, 2018
app pub date -
Sep 23, 2016
filing date -
Sep 23, 2016
priority date (Note) -
Published
status (Latency Note)
Importance
Abstract
Methods of modifying a dystrophin gene are disclosed, for restoring dystrophin expression within a cell having an endogenous frameshift mutation within the dystrophin gene. The methods comprising introducing a first cut within an exon of the dystrophin gene creating a first exon end, wherein said first cut is located upstream of the endogenous frameshift mutation; and introducing a second cut within an exon of the dystrophin gene creating a second exon end, wherein said second cut is located downstream of the frameshift mutation. Upon joining/ligation of said first and second exon ends dystrophin expression is restored, as the correct reading frame is restored. Reagents and uses of the method are also disclosed, for example to treat a subject suffering from muscular dystrophy.
First Claim
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