CF Foundation | Genetics Basics and an Introduction to Genetic Therapies for CF скачать в хорошем качестве

CF Foundation | Genetics Basics and an Introduction to Genetic Therapies for CF

This town hall is intended to introduce the cystic fibrosis community to new research efforts, including genetic therapies. This is the fourth in a four-part series of regional cystic fibrosis research town halls to familiarize the CF community with the clinical trial process, how clinical researchers protect participants’ safety and privacy, new areas of CF research, and how people with CF can get involved. The town hall was hosted by the Southeast Research Cooperative, which is made up of several research centers throughout the South that are committed to assisting with referrals for clinical trials and promotion of CF research throughout the region. Originally recorded October 5, 2023. 00:00 | Genetics Basics and an Introduction to Genetic Therapies for CF 04:11 | What is the role of genetics in CF? 05:29 | Understanding the Principles of Genetic-Based Therapies 10:35 | What is gene therapy? 11:38 | What are the four strategies to treat CF? 15:18 | Lindsey Guin, an adult with CF, discusses her participation in several research studies, including a messenger RNA therapy trial. 18:26| Did you have any concerns about entering into this kind of uncharted type of research study? 19:34 | Do you have any advice for people who may be contemplating involvement in a genetic therapy trial or a new type of research they have not participated in before to help ease their concerns or prepare them for participation? 22:47 | How are we moving genetic therapies into clinical trials for people with CF? 24:02 | How is the car analogy used to explain gene-based therapies for CF? 25:23 | How does gene delivery work? 30:34 | Disease example: Sickle Cell Disease (gene editing) 33:47 | What is the history of genetic-based therapies in CF? 37:50 | What industry programs are being funded by the CF Foundation? 38:35 | What are the ongoing or upcoming Phase 1/2 gene-based therapy studies? 40:13 | Who will be eligible for early trials? 41:13 | What are some helpful resources?