Entry - #500014 - MITOCHONDRIAL COMPLEX I DEFICIENCY, MITOCHONDRIAL TYPE 1; MC1DM1 - OMIM

# 500014

MITOCHONDRIAL COMPLEX I DEFICIENCY, MITOCHONDRIAL TYPE 1; MC1DM1



TEXT

A number sign (#) is used with this entry because of evidence that mitochondrial complex I deficiency mitochondrial type 1 (MC1DM1) is caused by mutation in the MTND3 gene (516002).

For a discussion of genetic heterogeneity of mitochondrial complex I deficiency, see 252010.


Clinical Features

Taylor et al. (2001) reported a 42-year-old man who had onset of migraine symptoms associated with flashing lights in his vision and right arm weakness at age 24 years. He subsequently developed myoclonus, seizures, cognitive decline, ataxia, peripheral neuropathy, eye movement abnormalities, and optic atrophy. Muscle biopsy showed a deficit (40% of controls) in complex I activity, but no ragged-red fibers.

McFarland et al. (2004) reported a patient with infantile encephalopathy and complex I deficiency. From birth, he was lethargic with hypotonia, areflexia, and muscle atrophy. Micrognathia and talipes equinovarus were noted.


Molecular Genetics

In a patient with mitochondrial complex I deficiency, Taylor et al. (2001) identified a heteroplasmic 10191T-C transition in the MTND3 gene (516002.0001) in skeletal muscle (77%) and blood (14%), as well as in his mother (3% in blood) and 2 unaffected sibs (barely detectable in blood).

McFarland et al. (2004) identified a mutation in the MTND3 gene (516002.0001) in a patient with infantile encephalopathy and complex I deficiency.


REFERENCES

  1. McFarland, R., Kirby, D. M., Fowler, K. J., Ohtake, A., Ryan, M. T., Amor, D. J., Fletcher, J. M., Dixon, J. W., Collins, F. A., Turnbull, D. M., Taylor, R. W., Thorburn, D. R. De novo mutations in the mitochondrial ND3 gene as a cause of infantile mitochondrial encephalopathy and complex I deficiency. Ann. Neurol. 55: 58-64, 2004. [PubMed: 14705112, related citations] [Full Text]

  2. Taylor, R. W., Singh-Kler, R., Hayes, C. M., Smith, P. E. M., Turnbull, D. M. Progressive mitochondrial disease resulting from a novel missense mutation in the mitochondrial DNA ND3 gene. Ann. Neurol. 50: 104-107, 2001. [PubMed: 11456298, related citations] [Full Text]


Creation Date:
Cassahndra L. Kniffin : 12/12/2018
Edit History:
carol : 12/13/2018

# 500014

MITOCHONDRIAL COMPLEX I DEFICIENCY, MITOCHONDRIAL TYPE 1; MC1DM1


DO: 0112101;  



TEXT

A number sign (#) is used with this entry because of evidence that mitochondrial complex I deficiency mitochondrial type 1 (MC1DM1) is caused by mutation in the MTND3 gene (516002).

For a discussion of genetic heterogeneity of mitochondrial complex I deficiency, see 252010.


Clinical Features

Taylor et al. (2001) reported a 42-year-old man who had onset of migraine symptoms associated with flashing lights in his vision and right arm weakness at age 24 years. He subsequently developed myoclonus, seizures, cognitive decline, ataxia, peripheral neuropathy, eye movement abnormalities, and optic atrophy. Muscle biopsy showed a deficit (40% of controls) in complex I activity, but no ragged-red fibers.

McFarland et al. (2004) reported a patient with infantile encephalopathy and complex I deficiency. From birth, he was lethargic with hypotonia, areflexia, and muscle atrophy. Micrognathia and talipes equinovarus were noted.


Molecular Genetics

In a patient with mitochondrial complex I deficiency, Taylor et al. (2001) identified a heteroplasmic 10191T-C transition in the MTND3 gene (516002.0001) in skeletal muscle (77%) and blood (14%), as well as in his mother (3% in blood) and 2 unaffected sibs (barely detectable in blood).

McFarland et al. (2004) identified a mutation in the MTND3 gene (516002.0001) in a patient with infantile encephalopathy and complex I deficiency.


REFERENCES

  1. McFarland, R., Kirby, D. M., Fowler, K. J., Ohtake, A., Ryan, M. T., Amor, D. J., Fletcher, J. M., Dixon, J. W., Collins, F. A., Turnbull, D. M., Taylor, R. W., Thorburn, D. R. De novo mutations in the mitochondrial ND3 gene as a cause of infantile mitochondrial encephalopathy and complex I deficiency. Ann. Neurol. 55: 58-64, 2004. [PubMed: 14705112] [Full Text: https://doi.org/10.1002/ana.10787]

  2. Taylor, R. W., Singh-Kler, R., Hayes, C. M., Smith, P. E. M., Turnbull, D. M. Progressive mitochondrial disease resulting from a novel missense mutation in the mitochondrial DNA ND3 gene. Ann. Neurol. 50: 104-107, 2001. [PubMed: 11456298] [Full Text: https://doi.org/10.1002/ana.1084]


Creation Date:
Cassahndra L. Kniffin : 12/12/2018

Edit History:
carol : 12/13/2018