NACFC 2023 | W05: Rescuing CFTR Premature Truncation Alleles
148 بار بازدید -
8 ماه پیش
-
This workshop provides an overview
This workshop provides an overview of the latest leading-edge technologies directed toward better cystic fibrosis treatment and basic electrophysiology, cellular biology, and protein chemistry related to understanding the cystic fibrosis transmembrane conductance regulator (CFTR). The session covers a range of topics, including nucleotide-based therapeutics, genome editing of the CF locus, and viral or non-viral strategies to maximize efficient, durable, and safe delivery of molecular interventions to respiratory tissues. By the end of the workshop, attendees will have gained valuable insights into the recent advancements in CF research, such as CFTR structure/activity, modulator response, and specific approaches to overcome nonsense, splice, and other refractory CFTR variants.
Originally recorded November 2, 2023.
Originally recorded November 2, 2023.
8 ماه پیش
در تاریخ 1402/08/29 منتشر شده
است.
148
بـار بازدید شده