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Discipline Group Session

CLIN

DG15--Emerging Topics in CF Genetics & Genetic Counseling

Friday, September 27, 2024
7:45 AM – 9:45 AM ET
Location: 252 B
Recorded Session
This session will review recent research updates with a focus on targeted therapies in CF across the lifespan. We will discuss off-label modulator use in pregnancy to treat affected fetuses, in vitro modulator responsiveness of CFTR variants with and without a currently approved modulator, and the current landscape of clinical trials for genetic therapies. This session is appropriate for genetic counselors and other clinicians incorporating genomics into CF care.

Learning Objectives:

  • Summarize logistical considerations and medical outcomes of off-label CFTR modulator use during pregnancy in fetuses with CF
  • Review the process of assessing CFTR variant responsiveness and potential impact on clinical care outcomes, with specific focus on newly published data on variants with and without an approved modulator
  • Discuss the current status and pipeline of clinical trials for genetic therapies (mRNA, gene replacement, and gene editing)