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2692705 : Pharmacological nonsense suppression therapy in the Rpe65-/- mouse model of Leber congenital amaurosis reverses the retinal deficits in vivo

Researchers

Presenter

  • Xia Wang

Principal Investigators

  • Olena Sivak

  • Kishor Wasan

  • Kevin Gregory-Evans

  • Cheryl Gregory-Evans

Medical Centers

  • University of Saskatchewan, Saskatoon, SK, Canada

  • University of British Columbia, Vancouver, British Columbia, Canada.

  • University of British Columbia, Vancouver, British Columbia, Canada.

Locations

  • Canada

Companies

  • N/A

Study Components

Therapeutic Area

  • Ophthalmology (OP)

  • Congenital disorder

Disease

  • N/A

Biomarkers

  • Cancerous inhibitor of protein phosphatase 2A

  • ETS transcription factor ERG

  • TNF Receptor Superfamily Member 1A

Drug/Treatment

  • Amlexanox

  • Ataluren

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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