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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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Glossar

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

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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

Related pages

Muscle diagram

Muscular dystrophy: how could stem cells help?

Factsheet
Verwandte Themen Muscle and Skeletal Diseases Muscular Dystrophy Rare Diseases Current and Potential Uses Factsheets
Cartoon showing a person looking at the contents of a cell

Gene Therapy Basics

Resource
Verwandte Themen Cell and Gene Fundamentals Gene Editing Gene Therapy
Cell Basics teaser image

What is gene and cell therapy?

Resource
Verwandte Themen Cell and Gene Fundamentals Cell Therapy Gene Editing Gene Therapy
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