FB2026_03 , released September 17, 2026
Human Disease Model Report: mitochondrial complex I deficiency, nuclear type 1
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General Information
Name
mitochondrial complex I deficiency, nuclear type 1
FlyBase ID
FBhh0000841
Overview

This report describes mitochondrial complex I deficiency, nuclear type 2 (MC1DN1); MC1DN1 exhibits autosomal recessive inheritance. The human gene implicated in this disease subtype is NDUFS4, a nuclear gene that encodes an accessory subunit of NADH:ubiquinone oxidoreductase (complex I), the first enzyme complex in the mitochondrial electron transport chain. There is a single fly ortholog, Dmel\ND-18, for which RNAi-targeting constructs have been generated.

The human NDUFS8 gene has not been introduced into flies.

Ubiquitous knockdown of Dmel\ND-18, effected by RNAi, results in a high percentage of pupal lethality; surviving adults exhibit reduced lifespan, severe feeding impairment, and locomotor defects. Larvae appear to ingest less food than control animals. In adults, the oxidative phsophorylation activity of Complex I is reduced, while the activity of Complex III is increased. Muscle-specific knockdown effected by RNAi results in comparable lifespan, feeding and locomotor defects. Pan-neuronal knockdown results in much milder phenotypes. Extensive physical interactions have been described for Dmel\ND-18; see below and in the ND-18 gene report.

[updated July 2019 by FlyBase; FBrf0222196]

Disease Summary Information
Parent Disease Summary: mitochondrial complex I deficiency, nuclear type
Symptoms and phenotype

Mitochondrial complex I deficiency causes a wide range of clinical disorders, ranging from lethal neonatal disease to adult-onset neurodegenerative disorders. Phenotypes include macrocephaly with progressive leukodystrophy, nonspecific encephalopathy, hypertrophic cardiomyopathy, myopathy, and liver disease. [from MIM:252010; 2016.08.12]

Specific Disease Summary: mitochondrial complex I deficiency, nuclear type 1
OMIM report

[MITOCHONDRIAL COMPLEX I DEFICIENCY, NUCLEAR TYPE 1; MC1DN1](https://omim.org/entry/252010)

Human gene(s) implicated

[NADH-UBIQUINONE OXIDOREDUCTASE Fe-S PROTEIN 4; NDUFS4](https://omim.org/entry/602694)

Symptoms and phenotype
Genetics

NDUFS4 is a nuclear gene. [NCBI Gene, NDUFS4 (human); 2018.07.11]

Mitochondrial complex I deficiency nuclear type 1 (MC1DN1) is caused by homozygous mutation in the nuclear-encoded NDUFS4 gene. [from MIM:252010; 2019.04.24]

Cellular phenotype and pathology
Molecular information

NDUFS4 encodes an accessory subunit of the mitochondrial membrane respiratory chain NADH dehydrogenase (complex I, or NADH:ubiquinone oxidoreductase). Complex I removes electrons from NADH and passes them to the electron acceptor ubiquinone. [Gene Cards, NDUFS4; 2018.07.11]

External links
Disease synonyms
Leigh syndrome, NDUFS4-related
MC1DN1
mitochondrial NADH dehydrogenase component of complex I, deficiency of
NADH:Q(1) oxidoreductase deficiency
NADH-coenzyme Q reductase deficiency
Ortholog Information
Human gene(s) in FlyBase
    Human gene (HGNC)
    D. melanogaster ortholog (based on DIOPT)
    Comments on ortholog(s)

    One to one: 1 human to 1 Drosophila.

    Other mammalian ortholog(s) used
      D. melanogaster Gene Information (1)
      Molecular function (GO)
        Gene Groups / Pathways
        Comments on ortholog(s)

        High-scoring ortholog of human NDUFS4 (1 Drosophila to 1 human). Dmel\ND-18 shares 55% identity and 76% similarity with the human gene.

        Orthologs and Alignments from DRSC
        DIOPT - DRSC Integrative Ortholog Prediction Tool - Click the link below to search for orthologs in Humans
        Other Genes Used: Viral, Bacterial, Synthetic (0)
          Summary of Physical Interactions (14 groups)
          protein-protein
          Interacting group
          Assay
          References
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          Alleles Reported to Model Human Disease (Disease Ontology) (1 alleles)
          Models Based on Experimental Evidence ( 1 )
          Modifiers Based on Experimental Evidence ( 0 )
          Allele
          Disease
          Interaction
          References
          Alleles Representing Disease-Implicated Variants
          Genetic Tools, Stocks and Reagents
          Sources of Stocks
          Contact lab of origin for a reagent not available from a public stock center.
          Bloomington Stock Center Disease Page
          Related mammalian, viral, bacterial, or synthetic transgenes
          Allele
          Transgene
          Publicly Available Stocks
          Selected Drosophila transgenes
          Allele
          Transgene
          Publicly Available Stocks
          RNAi constructs available
          Allele
          Transgene
          Publicly Available Stocks
          Selected Drosophila classical alleles
          Allele
          Allele class
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          Publicly Available Stocks
          References (4)