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FAQs

Why are some conditions more suitable for gene and cell therapies than others?
There is an approved treatment available for my condition - why can't I receive it?
How to look for approved medicines?
How are medicines developed and approved?

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Glossary

Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Antibody
Antigen
Base editors
Cas9 nuclease
Chimeric Antigen Receptor (CAR)
Chromosome
Clinical trial

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An Essential Partnership: A guide for charities working with industry

For case studies, see:

  • case study 2 on page 7
  • case study 7 page 14
  • case study 8 page 15
  • case study 22 page 26

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