Videos about genetic therapies
Q&A on genetic therapies - March 2023
Members of our involvement group quizzed three experts on CF genetic therapies in a video Question and Answer session. Watch the short recordings of three of the questions below.
A full recording is available on our YouTube channel. It is divided into sections (chapters), so you can go straight to the bit of the recording you’d like to hear about, or watch it all the way through.
Who could benefit from genetic therapies?
The researchers explain how groups of people who are unable to benefit from CFTR modulator medicines such as Kaftrio and Kalydeco (also known as Ivacaftor), and those that are on Kaftrio could see improvements with genetic therapies. They discuss whether and how a combination of both genetic therapies and CFTR modulators could bring additional (‘synergistic’) benefits to different types of CF.
(You can also watch this clip from 5m47s in the full recording)
What are the different types of genetic therapies?
The researchers explain that for people with some rare mutations there could be more than one type of genetic therapy that may be beneficial. For people with other rare mutations, there may be fewer options available.
Watch this animation to understand more about CF mutations.
They go on to talk about treatments being developed for: CF stop mutations; ‘ASO’ therapies CF splicing mutations; genetic therapies that could work for everyone, and the pros and cons of each.
They also discuss the difficulties for researchers in designing medicines to get to the right place.
(You can also watch this clip from 9m39s in the full recording)
Who will be able to take part in trials?
The researchers explain how people are selected to take part in clinical trials, the requirements are different for each trial. They also discuss whether people may be able to take a medicine later on in its development.
(You can also watch this clip from 28m2s in the full recording)
CF LIVE on genetic therapies
In June 2023 we hosted a Q&A session on CF genetic therapies for the CF community. Our expert panel were Professor Jane Davies, Dr Jamie Duckers and Dr Tim Lee. At the start of the event we asked the audience three poll questions and asked a member of our expert panel to address each one.
The answers to the three poll questions are given below, you can watch the full recording of the event on our YouTube channel. The list of Q&A is included in the video information, and also within the sections of the recording itself
Poll 1: True or False – Genetic therapies could be available for everyone regardless of the form of CF (or ‘CF mutation’)
Dr Jamie Duckers told us what genetic therapies are, and how medicines like Kaftrio are different to genetic therapies, using the example of making some flat pack furniture to explain the biology.
Poll 2: True or False – Genetic therapies offer a potential cure for CF.
Professor Jane Davies talks about her thoughts on whether genetic therapies are a cure. She discusses how long different types of genetic therapy are likely to last in the body (mRNA therapy, gene therapy and gene editing therapies), how soon they might be tested in clinical trials, and which parts of the body they will treat.
Poll 3: True or False – Taking genetic therapy treatments would modify my DNA
Dr Tim Lee explains whether and how different types of genetic therapy involve modifying DNA.
Genetic therapies playlist on YouTube
We've created a genetic therapies playlist on YouTube, where you can watch all of the videos listed on this page, plus some insights from the CF community and resources from other CF charities on this topic
Genetic therapies glossary
We’ve put together some definitions of words that you may come across as you learn more about genetic therapies. Where a word is in bold text, there is also a separate explanation of the word in this glossary.
You might also be interested in
General FAQs on genetic therapies
Read our Frequently Asked Questions on genetic therapies, beginning with explanations about genes and what genetic therapies are, and how they work.
Trials Tracker
Our Trials Tracker is regularly updated to show CF clinical trials happening across the UK
Genetic therapies glossary
We’ve put together some definitions of words that you may come across as you learn more about genetic therapies.