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):
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
