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PTAC/CFTR
Workshop

W05--Rescuing CFTR Premature Truncation Alleles: Basic Biology & Novel Therapeutic Strategies

Thursday, November 2, 2023
9:45 AM – 11:45 AM MST
Location:105 C (West)

Recorded Session

    Chair(s)

  • John Lueck

    Pharmacology and Physiology
    University of Rochester School of Medicine and Dentistry

  • Jianguo Chen, MD

    Cystic Fibrosis Research Center, University of Alabama at Birmingham.

This workshop will provide an overview of the latest leading-edge technologies directed toward better treatment of cystic fibrosis and basic electrophysiology, cellular biology, and protein chemistry related to understanding the cystic fibrosis transmembrane conductance regulator (CFTR).
The session will cover 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.

Learning Objectives:

  • Provide a summary of the latest updates on CFTR's fundamental electrophysiology, protein chemistry, and cellular biology that are necessary to understand the cystic fibrosis transmembrane conductance regulator.
  • Demonstrate the advancements and practical application of nucleotide-based therapies, gene editing techniques for CF, and both viral and non-viral delivery methods to maximize the efficient, durable, and safe dissemination of molecular interventions throughout the respiratory system.
  • Attendees are expected to gain knowledge on the most recent advancements in CF research, which consist of CFTR structure and activity, modulator response, and approaches to surmount refractory CFTR variants.

Presentations:

  • 9:45 AM – 11:45 AM MST
    W05.1- Development Of An Improved Adenine Base Editor To Correct W1282X-CFTR With Reduced Bystander Effects

    Speaker: Lucia Santos, PhD – BioISI – BioSystems and Integrative Sciences Institute, Faculty of Sciences, University of Lisbon

  • 9:45 AM – 11:45 AM MST
    W05.2- LUNAR®-CFTR mRNA replacement therapy restores CFTR expression and function in human bronchial epithelial cells

    Speaker: Javier Campos-Gomez, PhD – University of Alabama at Birmingham

  • 9:45 AM – 11:45 AM MST
    W05.3- Functional and RNA-level recovery of CFTR nonsense variants with NMD inhibition, readthrough, and ETI

    Speaker: Alice C. Eastman (she/her/hers) – Johns Hopkins School of Medicine

  • 9:45 AM – 11:45 AM MST
    W05.4- One ACE-tRNA sequence can functionally rescue the most prevalent CFTR nonsense mutations

    Speaker: Wooree Ko, MS – University of Rochester Medical Center

  • 9:45 AM – 11:45 AM MST
    W05.5- SPL84, Antisense oligonucleotide modulating the 3849+10kb C-T splicing defect, efficiently penetrates and restores CFTR function when treated through the apical side of primary HBE cells

    Speaker: Efrat Ozeri-Galai, PhD – splisense