31st Annual North American Cystic Fibrosis Conference

W12 - W12: CFTR: Toward a One Time Cure: Technologies & Editing

Nov 3, 2017 2:00pm ‐ Nov 3, 2017 3:20pm


Description

This session will highlight new technologies and gene editing strategies that will move the field towards a one time cure.

Learning Objectives:

  1. Describe the potential power of CRISPR CAS9 based gene editing platforms for drug development and a one time cure
  2. Discuss the advantages of engineered tRNA for suppression of CFTR nonsense mutations
  3. Test the efficiency of oligonucleotide mediated therapy to restore CFTR function

Note: Speaker Adele Ricciardi's presentation was not recorded at their request.

Workshop Chair(s):

Workshop Speaker(s):

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