Experts answer your questions 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.

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.

Our genetic therapy glossary explains some words used in talking about this type of therapy, including words used in these explanations.

Look up a word in our genetic therapies glossary

CF LIVE Genetic therapies for CF: any questions?

The polls above are clips from an hour-long recording of Genetic therapies Q&A event, part of our CF LIVE series. You can watch the whole recording again.

From approximately 23 minutes into the recording, the panel took questions from the audience. The questions asked are given below (with approximate timings in the recording given in brackets).

  • When do we expect genetic therapies to be widely available (~23m30s)
  • When will the BI gene therapy trial start (~29m)
  • Is there any research into stem cells, for example using those from the umbilical cord? (~34m)
  • What form would a genetic therapy be given eg pills, nebulizers etc (~37m)
  • Is there a specific age to participate in trials? (~39m)
  • How do you find out about clinical trials, do I have to live near a trial centre? (~41m45s)
  • How do you decide which trial to take part in? (~44m20s)
  • My daughter isn’t benefitting from Kaftrio, could she take part in a genetic therapy trial? (~46m30s)
  • Where are the specialist centres running these trials? (~48m30s)
  • If a person with CF had NTM in the past, would this prevent them from taking part in genetic therapy trials? (~49m30s)
  • Would liver cirrhosis be an exclusion criteria for genetic therapy trial? (~52m40s)
  • I’m from Malta, would I be able to take part in one of these trials? (54m20s)

Find out more about CF genetic therapies

CF genetic therapies

Find out what they are, whether they might benefit you and learn more about genetic therapy clinical trials. Watch a Q&A session and read our general frequently asked questions on CF genetic therapies.

Genetic therapies glossary

We’ve put together some definitions of words that you may come across as you learn more about genetic therapies.

FAQs on genetic therapy clinical trials

Read our Frequently Asked Questions on genetic therapy clinical trials, topics range from can I take part, to what will be involved.

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