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FAQs

Why are animal models used when testing the safety of a new therapy?
What role do preclinical and clinical trials play in ensuring patient safety?
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

Accelerated assessment
Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Advanced Therapy Investigational Medicinal Product (ATIMP)
Advanced Therapy Medicinal Product (ATMP)
Advertising 
Antibody
Antigen
Base editors

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Current and Potential Uses

Resources & Factsheets

EuroGCT Resource
EuroGCT stock DNA graphic

Why are some conditions more suitable for gene and cell therapies than others?

Related Topics Cell Therapy Clinical Trials Current and Potential Uses Disease Modelling Ethics and Society Gene Editing Gene Therapy Methods and Tools Patient Involvement Policy & Regulation Research
English

European Reference Networks

Related Topics Current and Potential Uses Ethics and Society Policy & Regulation
English
EuroGCT Resource
EuroGCT DNA image

Using 'reprogrammed' cells to model diseases

Related Topics Current and Potential Uses Disease Modelling iPS cells Methods and Tools Organoids Reprogramming Tissue Engineering
English
EuroGCT Resource
Photograph of a healthy liver

Chronic liver disease: how could gene and cell therapy help?

Related Topics Liver Disease Cell Therapy Current and Potential Uses Factsheets iPS cells Organoids Tissue Engineering
English
EuroGCT Resource
Laboratory-grown nerve cells, shown with fluorescent green dye against a black background

Parkinson's Disease: How could gene and cell therapy help?

Related Topics Neurological Disorders Parkinson's Disease Cell Therapy Clinical Trials Current and Potential Uses Factsheets Gene Therapy iPS cells Reprogramming
European Medicines Agency logo

Compassionate use

Related Topics Rare Diseases Cell Therapy Clinical Trials Current and Potential Uses Gene Therapy Patient Involvement Policy & Regulation Unproven Treatments
Cartoon graphic of two figures hugging. One wears an insulin patch on their arm.

Connecting young people with Type 1 Diabetes through science

Related Topics Diabetes Cell Therapy Current and Potential Uses Embryonic Stem Cells Patient Involvement Reprogramming Research Stem Cell News
Cartoon image of a chart headed 'Patient Examination'

Identifying Genetic Disease

Related Topics Current and Potential Uses Ethics and Society Methods and Tools
Screenshot from ASGCT's video 'Gene Therapy Approaches'. Two cartoon figures look at text listing gene therapy techniques: Gene Addition, RNA Therapy, Gene Silencing, Cell Therapy, Gene Editing

Gene Therapy Approaches

Related Topics Cell and Gene Fundamentals Cell Therapy Clinical Trials Current and Potential Uses Gene Editing Gene Therapy Methods and Tools Reprogramming
English
Image of intestinal epithelial cells

Short bowel syndrome: how can gene and cell therapy help?

Related Topics Bowel Disease Current and Potential Uses Disease Modelling Factsheets iPS cells Tissue Engineering
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