Description

This workshop highlights advancements in new and emerging technologies progressing towards a cure. Discussions will include development of tools/strategies focused on rare and nonsense CFTR variants with translatable relevance to all CFTR mutations.

Learning Objectives:

• Summarize molecular defects attributable to CFTR premature truncation codons, including the role of nonsense mediated decay and its potential as a therapeutic target.
• Compare approaches for CFTR gene repair or replacement in primary airway epithelia.
• Evaluate state-of-the-art directed differentiation of airway progenitor cells from pluripotent source material.

Note: Speaker Youngjin Kim's presentation was not recorded at his request.

Workshop Chair(s):

• John Mahoney, Ph.D., Group Leader, Scientist II
• Kathryn E. Oliver, Ph.D., Postdoctoral Fellow

Workshop Speaker(s):

• Shingo Suzuki, Ph.D., Postdoctoral fellow, UC, San Francisco
• Youngjin Kim, B.S., MD/PhD student
• Farruk Kabir, Ph.D.
• Andras Rab, M.D., Assistant Professor
• Afroditi Avgerinou, B.Sc., M.Sc., Miss