Description

Note: This was a special poster discussion workshop session and its content was not suitable for recording. Significant advances in gene delivery and gene editing are meeting major progress in pharmacological correction of specific CFTR mutations. These advances have synergized to allow researchers to develop novel cellular and animal based tools in the study of CFTR. These tools are being developed into creative screens to uncover new methods of restoring CFTR function as well as characterizing the mechanisms of known CFTR modulators, correctors and potentiators. This session will provide a broad overview and discussion of multiple approaches to correcting CFTR gene defects. Educational Objectives: Evaluate the next generation of CFTR modulators, correctors and potentiators. Discuss the latest advances in CFTR gene editing and gene therapy. Discuss the most recent advances in cellular and animal models for assessing CFTR function and identifying novel therapeutic targets.

Speaker(s):

• Venkateshwar Mutyam
• Hermann Bihler, Ph.D.
• Fredrick van Goor, Ph.D., Sr. Research Fellow, Biology, Vertex Pharmaceuticals, Inc.
• Neeraj Vij, Ph.D.
• Ashvani Singh, Ph.D.
• Nicholas Gill, B.A.
• Maria Montiel, Ph.D.
• Patrick Harrison, Ph.D., Senior Lecturer
• Patrick Sinn, Ph.D., Associate Professor
• Benjamin T. Kopp, M.D., M.P.H, FAAP, Assistant Professor of Pediatrics, Nationwide Children's Hospital
• Jyoti Sharma, M.S., Student
• Viral Shah, BS, Graduate Student, University of Iowa Carver College of Medicine
• Annelotte Vonk
• Bob Scholte, Ph.D.