P171: Developing an approach to screening rare genetic diagnoses for amenability to bespoke genetic therapy development

• David Cheerie,
• Marlen Lauffer,
• Danique Beijer,
• Matthis Synofzik,
• Annemieke Aartsma-Rus,
• Michael Szego,
• Kimberly Amburgey,
• Brian Kalish,
• James Dowling,
• Gregory Costain

Affiliations

David Cheerie

Genetics and Genome Biology, SickKids Research Institute, Toronto, ON, Canada

Marlen Lauffer

Dutch Center for RNA Therapeutics, Leiden University Medical Center, The Netherlands

Danique Beijer

Hertie-Institute for Clinical Brain Research, University of Tübingen, Germany

Matthis Synofzik

Hertie-Institute for Clinical Brain Research, University of Tübingen, Germany

Annemieke Aartsma-Rus

Dutch Center for RNA Therapeutics, Leiden University Medical Center, The Netherlands

Michael Szego

Joint Centre for Bioethics, University of Toronto, Toronto, ON, Canada

Kimberly Amburgey

Genetics and Genome Biology, SickKids Research Institute, Toronto, ON, Canada

Brian Kalish

Neurosciences and Mental Health, SickKids Research Institute, Toronto, ON, Canada

James Dowling

Genetics and Genome Biology, SickKids Research Institute, Toronto, ON, Canada

Gregory Costain

Genetics and Genome Biology, SickKids Research Institute, Toronto, ON, Canada