@frattalab : Want to deliver gene therapies broadly, but activate them only in diseased neurons? TDP-REG "hacks" TDP cryptic mis-splicing to drive expression only WHEN and WHERE needed! Less toxicity, wider therapeutic window, new options for sporadic ALS therapies. biorxiv.org/content/150.110… • TwiCopy

FrattaLab

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FrattaLab @UCLIoN working on #RNA biology of #ALS and #SBMA and #Kennedy's disease

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linkhttps://www.frattalab.org calendar_today01-11-2019 11:35:16

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FrattaLab

@frattalab

2 years ago

Want to deliver gene therapies broadly, but activate them only in diseased neurons? TDP-REG "hacks" TDP cryptic mis-splicing to drive expression only WHEN and WHERE needed! Less toxicity, wider therapeutic window, new options for sporadic ALS therapies. biorxiv.org/content/10.110…

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