ID

45211

Beschrijving

Principal Investigator: Tadafumi Kato, MD, PhD, RIKEN Brain Science Institute, Saitama, Japan MeSH: Ophthalmoplegia, Chronic Progressive External https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs000392 Progressive external ophthalmoplegia (PEO) is an inherited mitochondrial disease that follows either autosomal dominant or recessive forms of inheritance (adPEO or arPEO). AdPEO is a genetically heterogeneous disease and several genes including *POLG1* and *C10orf2*/Twinkle have been identified as responsible genes. On the other hand, *POLG1* was the only established gene causing arPEO with mitochondrial DNA deletions. We previously reported a case of PEO with unidentified genetic etiology. The patient was born of a first-cousin marriage. Therefore, the recessive form of inheritance was suspected. To identify the disease causing variant in this patient, we subjected the patient's DNA to whole-exome sequencing and narrowed down the candidate variants using public data and runs of homozygosity analysis.

Link

dbGaP study = phs000392

Trefwoorden

  1. 02-08-22 02-08-22 - Simon Heim
  2. 12-10-22 12-10-22 - Adrian Schulz
Houder van rechten

Tadafumi Kato, MD, PhD, RIKEN Brain Science Institute, Saitama, Japan

Geüploaded op

12 oktober 2022

DOI

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Licentie

Creative Commons BY 4.0

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dbGaP phs000392 Exome Sequencing of arPEO

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Eligibility Criteria

Name
Type
Description | Question | Decode (Coded Value)
Datatype
Alias
Item Group
Inclusion and exclusion criteria
Single case of progressive external ophthalmoplegia was included.
Item
Single case of progressive external ophthalmoplegia was included.
boolean
C1512693 (UMLS CUI [1,1])
C0162674 (UMLS CUI [1,2])

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