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Gene (Targeted Genetic Mutation) (N/A)

Target: N/A
Molecular classification: Gene, DNA sequence

Overview

Genetic mutation correction aims to precisely repair or eliminate disease-causing mutations in DNA. This involves using genome editing technologies to target specific genomic locations and induce DNA double-strand breaks, followed by cellular repair mechanisms to correct the mutation. Strategies include CRISPR-Cas9, TALENs, ZFNs, and mito-TALENs for mitochondrial DNA. The ultimate goal is to restore normal gene function and prevent or treat genetic diseases.

Other names: Pathogenic Genetic VariantDisease-causing MutationMutated GeneTargeted Mutation

Mechanism of action

Genome editing technologies (CRISPR-Cas9, TALENs, ZFNs) to induce DNA double-strand breaks and promote homology-directed repair (HDR) or non-homologous end joining (NHEJ). Single-stranded oligonucleotides (ssODNs) as templates for HDR. mito-TALENs/ZFNs for elimination of mutated mitochondrial DNA.

Biological functions

Gene expressionProtein synthesisCellular function (dependent on the specific gene)

Disease associations

Monogenic disordersGenetic diseasesCancer (in some cases)Mitochondrial diseases

Safety considerations

Off-target effects (unintended edits at other genomic locations)Immunogenicity of gene editing componentsMosaicism (incomplete correction in all cells)Unintended consequences of DNA repair mechanismsGermline editing concerns

Biomarkers

Specific DNA sequence of the mutationExpression levels of the corrected geneFunctional assays of the corrected protein

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