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Disease-causing gene
- Molecular classification
- Gene, Protein, Enzyme, Receptor, Structural protein, Transcription factor, Other
01
Overview
The disease-causing gene that is being targeted for replacement or supplementation via adeno-associated viral (AAV) vector delivery. The specific gene and its function depend on the disease being treated. AAV vectors are engineered to deliver a functional copy of the gene to target cells, compensating for the defective or missing endogenous gene. The cell-surface molecules (receptors) that mediate uptake of specific AAV serotypes are also considered targets to allow efficient gene delivery. Examples include fibroblast/hepatocyte growth factor receptor, integrins αVβ5/α5β1, epidermal growth factor receptor, platelet-derived growth factor receptor and AAVR.
- Other names
- Defective geneMissing gene
02
Mechanism of action
Gene replacement or supplementation via AAV vector delivery
03
Biological functions
Varies depending on the specific gene. Examples: Metabolism, Signal transduction, DNA replication, Protein synthesis, Cell structure
04
Disease associations
Genetic disordersMonogenic diseasesNeurological disordersHemophiliaKidney diseaseOther
05
Safety considerations
Pre-existing immunity to AAVLiver toxicityOff-target effectsInsertional mutagenesis (rare)Immune response to the therapeutic protein
06
Biomarkers
Expression level of the therapeutic genePresence of neutralizing antibodies against AAVLiver enzyme levels (for systemic delivery)
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