Genome Sequencing Identifies the Pathogenic Variant Missed by Prior Testing in an Infant with Marfan Syndrome.

TitleGenome Sequencing Identifies the Pathogenic Variant Missed by Prior Testing in an Infant with Marfan Syndrome.
Publication TypeJournal Article
Year of Publication2019
AuthorsWojcik, MH, Thiele, K, Grant, CF, Chao, K, Goodrich, J, O'Donnell-Luria, A, Lacro, RV, Tan, W-H, Agrawal, PB
JournalJ Pediatr
Volume213
Pagination235-240
Date Published2019 Oct
ISSN1097-6833
Abstract

We describe an infant with a phenotype typical of early onset Marfan syndrome whose genetic evaluation, including Sanger sequencing and deletion/duplication testing of FBN1 and exome sequencing, was negative. Ultimately, genome sequencing revealed a deletion missed on prior testing, demonstrating the unique utility of genome sequencing for molecular genetic diagnosis.

DOI10.1016/j.jpeds.2019.05.029
Alternate JournalJ. Pediatr.
PubMed ID31235381
PubMed Central IDPMC6765408
Grant ListR01 AR068429 / AR / NIAMS NIH HHS / United States
K12 HD052896 / HD / NICHD NIH HHS / United States
UM1 HG008900 / HG / NHGRI NIH HHS / United States
U19 HD077671 / HD / NICHD NIH HHS / United States
T32 GM007748 / GM / NIGMS NIH HHS / United States
R01 EY027421 / EY / NEI NIH HHS / United States