Target intelligence / Profile preview

Nuclease MG29-1 (MG29-1)

Target: MG29-1
Molecular classification: Enzyme, CRISPR-associated nuclease, Type V CRISPR nuclease

Overview

Nuclease MG29-1 is a novel, metagenomics-derived Type V CRISPR-associated nuclease engineered for high specificity and efficiency in genome editing. It utilizes guide RNAs (gRNAs) to direct its activity to specific DNA sequences, inducing double-strand breaks (DSBs) and enabling gene knockout or insertion. It is being developed for therapeutic applications, including in vivo and ex vivo gene editing for monogenic diseases such as hemophilia A.

Mechanism of action

RNA-guided DNA cleavage

Biological functions

Genome editingDNA cleavageTargeted gene knockoutTargeted gene insertion

Disease associations

Genetic disordersHemophilia A

Safety considerations

Off-target effectsImmunogenicity

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