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Gene Therapy
- Molecular classification
- Not applicable
01
Overview
Gene therapy is a medical technique that aims to treat or prevent disease by modifying a person's genetic material. This can involve replacing, inactivating, or introducing genes within an individual's cells to correct underlying genetic problems or provide new functions. The approach targets the root cause of diseases at the DNA level rather than just managing symptoms.
- Other names
- Genetic TherapyGene transfer
02
Mechanism of action
Replacing a disease-causing gene with a healthy copy; inactivating a malfunctioning gene; introducing new or modified genes to help fight disease
03
Biological functions
Treatment of genetic diseasesDisease preventionGene editingGene insertionGene silencing
04
Disease associations
Genetic disordersCancerEye diseasesNeuromuscular disordersInfectious diseases
05
Safety considerations
Off-target effectsImmune responses against vectorsInsertional mutagenesisLong-term safety
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