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PTAC/CFTR
Symposium

S25--Genetic Repair of CFTR: Advancement of Superexon, Editing, & Related Technologies

Saturday, November 4, 2023
9:30 AM - 11:30 AM MST
Location:103 AB (West)

Recorded Session

In this symposium, we will explore how genetic therapies are being used to repair or augment mutated CFTR. We will also explore emerging technologies with applications for CFTR editing.

Learning Objectives:

  • Summarize the current state of the art in genetic therapies, with a focus on gene editing approaches.
  • Describe recent technological developments in this area.
  • Illustrate how gene editing approaches are being applied to CF.

Presentations:

  • 9:30 AM - 11:30 AM MST
    S25.1- Genetic Therapy For Almost All CFTR Mutations – The Super Exon Approach

    Speaker: Hillary Valley, PhD (she/her/hers) – Cystic Fibrosis Foundation

  • 9:30 AM - 11:30 AM MST
    S25.2- Prime Editing to correct genetic hotspots and prevalent mutations causing Cystic fibrosis

    Speaker: Jeremy Duffield (he/him/his) – Prime Medicine

  • 9:30 AM - 11:30 AM MST
    S25.3- Diverse CFTR Variants and Considerations for CFTR Gene Editing Strategies

    Speaker: Sriram Vaidyanathan, PhD – Nationwide Children's Hospital

  • 9:30 AM - 11:30 AM MST
    S25.4- Designing new carriers for nebulized mRNA delivery to the lung

    Speaker: Philip J. Santangelo, PhD (he/him/his) – Emory University