Partial paternal uniparental disomy (UPD) of chromosome 1 in a patient with Stargardt disease
2007

Partial paternal uniparental disomy of chromosome 1 in a patient with Stargardt disease

Sample size: 77 publication Evidence: moderate

Author Information

Author(s): R. Riveiro-Alvarez, D. Valverde, I. Lorda-Sanchez, M. J. Trujillo-Tiebas, D. Cantalapiedra, E. Vallespin, J. Aguirre-Lamban, C. Ramos, C. Ayuso

Primary Institution: Fundacion Jimenez Diaz

Hypothesis

This study aims to describe a form of non-mendelian inheritance in a patient with Stargardt disease.

Conclusion

Recessive Stargardt disease can rarely be inherited from only one unaffected carrier parent in a non-mendelian manner.

Supporting Evidence

  • A patient with Stargardt disease was found to have a homozygous mutation in the ABCA4 gene.
  • Haplotype analysis suggested that no maternal ABCA4 allele was transmitted to the patient.
  • The cytogenetic study revealed a normal female karyotype.
  • Further evaluation showed the patient had a normal dosage for both copies of the ABCA4 gene.

Takeaway

Sometimes, a child can inherit a disease from just one parent who doesn't show any symptoms, which is unusual.

Methodology

DNA from the patient and relatives was analyzed for variants in all 50 exons of the ABCA4 gene using microarray screening and confirmed by direct sequencing.

Limitations

The study could not assess other tissues than peripheral lymphocytes due to loss of contact with the family.

Participant Demographics

The patient was a 40-year-old Caucasian female born to nonconsanguineous, healthy parents.

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