Gene Therapy Explained

Choose a language

Gene therapy for sickle cell and beta thalassaemia

Powered by Real Patient Experiences.

Clear guidance on the gene therapy journey for sickle cell disease and beta thalassaemia, from the first conversation to long-term follow-up, alongside the people who have already been through it.

This site is completely non-profit.

See the whole journey
Who are you here for?

This changes what you see first, and how much medical detail comes first. You can change it or turn it off at any time.

Your choice is kept on this device only. It is not linked to who you are, and nothing is sent anywhere.

The whole path, in order

Most people arrive somewhere in the middle. Wherever you are, you can see what is behind you and what comes next.

The gene therapy path

Choose a stage to see where it sits on the path.

Deciding

Stage 1 of 7

This stage

After this: Eligibility and assessment, Stem cell collection, Conditioning, Hospital admission, Recovery, Long-term follow-up

Read more about DecidingWalk through the whole journey

Eligibility and assessment

Stage 2 of 7

This stage

Before this: Deciding

After this: Stem cell collection, Conditioning, Hospital admission, Recovery, Long-term follow-up

Read more about Eligibility and assessmentWalk through the whole journey

Stem cell collection

Stage 3 of 7

This stage

Before this: Deciding, Eligibility and assessment

After this: Conditioning, Hospital admission, Recovery, Long-term follow-up

Read more about Stem cell collectionWalk through the whole journey

Conditioning

Stage 4 of 7

This stage

Before this: Deciding, Eligibility and assessment, Stem cell collection

After this: Hospital admission, Recovery, Long-term follow-up

Read more about ConditioningWalk through the whole journey

Hospital admission

Stage 5 of 7

This stage

Before this: Deciding, Eligibility and assessment, Stem cell collection, Conditioning

After this: Recovery, Long-term follow-up

Read more about Hospital admissionWalk through the whole journey

Recovery

Stage 6 of 7

This stage

Before this: Deciding, Eligibility and assessment, Stem cell collection, Conditioning, Hospital admission

After this: Long-term follow-up

Read more about RecoveryWalk through the whole journey

Long-term follow-up

Stage 7 of 7

This stage

Before this: Deciding, Eligibility and assessment, Stem cell collection, Conditioning, Hospital admission, Recovery

Read more about Long-term follow-upWalk through the whole journey

Or walk through the whole journey, step by step

Written from published sources

This page is written from published guidance and research by regulators, the NHS and peer-reviewed journals. Every source is listed at the bottom of the page so you can check it yourself.

Lived experience

Written by a person describing their own life. It is not clinical guidance and has not been reviewed as such.

Talking to a person

Asking for support, and sharing what you have been through, are two different things. Choose the one that fits.

Would you like to speak to someone who has been through it?

A page can only go so far. If you would rather talk to a person, you can ask. You will be contacted first. Nothing is published.

Request a conversation

Share your experience

If you have been through it, you can say how it was, and choose a nickname or your real name to sit with those words. If you are willing to talk to someone who is still deciding, there is a question about whether your contact details may be kept. Nothing is published.

Share your experience

This platform is in development. Guidance is being written and reviewed, and no page is published until a named clinician has approved it.