CF Foundation | Research Update: Genetic Therapies for Cystic Fibrosis скачать в хорошем качестве

CF Foundation | Research Update: Genetic Therapies for Cystic Fibrosis

Genetic therapies, including RNA therapy and gene therapy, could potentially work for any person with cystic fibrosis, including people with nonsense and rare mutations. During our webinar, Kara Foshay, PhD, and Heather Hathorne, PhD, RRT, CCRC, share an overview of genetic therapies, starting from the basics: How do genetic therapies work in the body? How are they different from existing therapies? What kind of benefits could they provide? You’ll also learn about genetic therapy clinical trials, including how to find enrolling trial opportunities and what participation in a clinical trial can entail. This webinar was moderated by Schyler Kline, an adult with CF who has participated in a genetic therapy clinical trial. Originally recorded March 20, 2025. 0:00:00 | Research Update: Genetic Therapies for Cystic Fibrosis 04:21 | Understanding CFTR mutations and the flow of genetic information 07:15 | What types of genetic therapies are being developed for CF? 09:12 | What is the status of CF genetic therapy clinical trials? 10:45 | What are the differences between mRNA therapy and gene therapy? 13:04 | Why is delivering genetic therapies to the lungs so challenging? 16:12 | What are some of the safety challenges of genetic therapies? 19:51 | When is a genetic therapy ready to move from the lab into clinical trials? 26:22 | What to expect when participating in a genetic therapy clinical trial 28:45 | What is the informed consent process? 30:49 | How is your safety protected in a clinical trial? 32:00 | What are the differences between the genetic therapy clinical trials that are currently enrolling people with CF? 34:52 | How can you find clinical trial opportunities and get involved in research? 37:55 | Can you participate in a clinical trial at a location other than your own care center? 41:30 | What is the REACH study? 45:02 | What are the key concepts people with CF need to understand prior to participating in a genetic therapy clinical trial? 45:57 | Are genetic therapies a one-time fix, and will they benefit other organs outside of the lungs? 47:32 | Can you explain the difference between genetic therapies and CFTR modulators? 48:32 | How close are CF genetic therapies to completing Phase 2 clinical trials? 50:03 | With a small population of people with CF who can participate in genetic therapy trials, won’t it take a long time to complete this research? 50:45 | As a clinical trial participant, why did you select one type of genetic therapy trial over another? 52:04 | Do these clinical trials need non-CF healthy volunteers for safety? 52:52 | What mechanism helps a viral vector make it through mucus into lung cells? 53:38 | How can a person with CF know the best time to enroll in a genetic therapy study? 54:57 | Are any genetic therapy trials happening internationally? 55:27 | If you participate in a genetic therapy trial, are you excluded from future trials? 56:16 | Closing thoughts: What are you most excited about in this area of research? Additional resources: Clinical Trial Finder | https://apps.cff.org/trials/finder Clinical Trial Navigator | https://www.cff.org/research-clinical... Research Into Genetic Therapies | https://www.cff.org/research-clinical... Patient Safety in CF Clinical Trials | https://www.cff.org/research-clinical...