We can repair and replace genes in the lab, but what are the challenges of doing this in people? There is so much diversity in cystic fibrosis mutations — more than 1,700 different ones — that we may need to tailor genetic therapies to ensure everyone has a treatment.
At this session, hear from two experts in the genetic research field, Mitchell Drumm, PhD, and Rebecca Darrah, PhD, MS. Viewers can also learn about different types of mutations – for example, splice and nonsense – and the innovative new genetic therapies (mRNA therapy, gene therapy, and gene editing) currently in development to ensure all people with CF can benefit.
Originally recorded April 26, 2023.
00:00:00 | Genetics Therapies: The Latest Innovations and Potential Benefits
00:02:58 | Rena Borrows shares her CF story
00:07:35 | Meet Rebecca Darrah, PhD, MS and receive an introduction to CF genetics
00:11:30 | Rebecca Darrah discusses why a gene is a recipe
00:13:35 | Learning about nonsense mutations as it relates to a recipe
00:14:19 | Learning about splice mutations as it relates to a recipe
00:16:59 | Approaches to genetic therapies
00:19:19 | The current CFTR modulator landscape
00:21:47 | Introduction of Mitchell Drumm, PhD
00:23:03 | How to create an acceptable genetic recipe
00:26:50 | What is the difference between gene editing vs. gene therapy?
00:32:03 | What is the difference between RNA editing vs. mRNA therapy?
00:34:10 | Learn about protein repair, replace, or substitute
00:35:36 | Focusing on the lungs with gene editing, gene therapy, RNA editing, and mRNA therapy
00:42:38 | Current clinical trials on gene editing, gene therapy, RNA editing, and mRNA therapy
00:43:37 | Variations of CFTR mutations
00:44:32 | What is the timeline of CF gene therapy clinical trials?
00:45:21 | What would mRNA replacement in the clinic look like?
00:50:15 I Overview of the CF Foundation genetic therapies pipeline
00:52:08 I Panelists share genetic therapies resources
