Target intelligence / Profile preview

Intron-encoded endonuclease I-SceI (I-SceI) (I-SceI)

Target
I-SceI
Molecular classification
Enzyme, Homing endonuclease, LAGLIDADG family meganuclease, Deoxyribonuclease
01

Overview

Intron-encoded endonuclease I-SceI, commonly known as I-SceI meganuclease, is a site-specific homing endonuclease originally identified in the mitochondria of the yeast Saccharomyces cerevisiae [1]. It belongs to the LAGLIDADG family of proteins and is renowned for its ability to recognize and cleave a highly specific 18-base pair asymmetric DNA sequence (5'-TAGGGATAACAGGGTAAT-3'), a sequence so rare it is statistically unlikely to occur naturally in the human genome [2]. This extreme specificity allows I-SceI to serve as a precise molecular scalpel, inducing targeted double-strand breaks (DSBs) that activate cellular DNA repair mechanisms like homology-directed repair (HDR) or non-homologous end joining (NHEJ) [3][4]. While newer technologies like CRISPR/Cas9 offer greater flexibility, I-SceI remains a critical gold standard for studying DNA repair kinetics and developing gene correction therapies for genetic disorders [5]. In a therapeutic context, I-SceI is utilized as a component of gene editing platforms to disrupt viral DNA or facilitate gene replacement, though its clinical use is limited by the requirement for its specific recognition site [6]. Safety considerations include potential off-target effects at near-match sequences and the immunogenicity of the yeast-derived protein when introduced into human patients [4][5].

Other names
Omega-nucleaseScAlEndo.SceISceI meganucleaseIntron-encoded endonuclease 1
02

Mechanism of action

Induces site-specific DNA double-strand breaks (DSBs) at an 18-base pair recognition sequence, triggering cellular DNA repair mechanisms such as non-homologous end joining (NHEJ) or homology-directed repair (HDR) to achieve targeted genome modification [1][2][4].

03

Biological functions

DNA cleavage [1]Double-strand break induction [2]Gene conversion [1]Homing [3]Genome modification [5]
04

Disease associations

Genetic disorder [5]Infection [6]Cancer [2]
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Safety considerations

Off-target DNA cleavage [4]Immunogenicity of yeast-derived protein [5]Genotoxicity [4]Chromosomal translocation [6]Delivery efficiency [5]
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Biomarkers

DNA cleavage efficiency [2]Indel frequency [4]Homology-directed repair (HDR) rate [5]GFP expression in reporter assays [2]

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