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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?
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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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Potential Gene Therapies for Duchenne Muscular Dystrophy

Related topics:
Muscle and Skeletal Diseases Muscular Dystrophy Gene Editing Gene Therapy Research
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Action Duchenne have created a page on gene therapy for Duchenne Muscular Dystrophy. Here you will find an explanation of the underlying principles of gene therapy for DMD, and an overview of the different avenues of research being explored.

Action Duchenne: Gene Therapy

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Copyright Action Duchenne - Registered Charity No 1101971 - Scottish Charity No SC043852

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