Diagnosis of TBC1D32-associated conditions: Expanding the phenotypic spectrum of a complex ciliopathy.


Journal

American journal of medical genetics. Part A
ISSN: 1552-4833
Titre abrégé: Am J Med Genet A
Pays: United States
ID NLM: 101235741

Informations de publication

Date de publication:
05 2023
Historique:
revised: 25 01 2023
received: 22 07 2022
accepted: 03 02 2023
medline: 10 4 2023
pubmed: 25 2 2023
entrez: 24 2 2023
Statut: ppublish

Résumé

Exome sequencing is a powerful tool in prenatal and postnatal genetics and can help identify novel candidate genes critical to human development. We describe seven unpublished probands with rare likely pathogenic variants or variants of uncertain significance that segregate with recessive disease in TBC1D32, including four fetal probands in three unrelated pedigrees and three pediatric probands in unrelated pedigrees. We also report clinical comparisons with seven previously published patients. Index probands were identified through an ongoing prenatal exome sequencing study and through an online data sharing platform (Gene Matcher™). A literature review was also completed. TBC1D32 is involved in the development and function of cilia and is expressed in the developing hypothalamus and pituitary gland. We provide additional data to expand the phenotype correlated with TBC1D32 variants, including a severe prenatal phenotype associated with life-limiting congenital anomalies.

Identifiants

pubmed: 36826837
doi: 10.1002/ajmg.a.63150
pmc: PMC10204718
mid: NIHMS1896050
doi:

Substances chimiques

TBC1D32 protein, human 0
Adaptor Proteins, Signal Transducing 0

Types de publication

Review Journal Article Research Support, Non-U.S. Gov't Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

1282-1292

Subventions

Organisme : NICHD NIH HHS
ID : R01 HD105868
Pays : United States

Informations de copyright

© 2023 Wiley Periodicals LLC.

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Auteurs

Sarah C Harris (SC)

Department of Obstetrics and Gynecology, Prisma Health, University of South Carolina Greenville, Greenville, South Carolina, USA.

Karen Chong (K)

The Prenatal Diagnosis and Medical Genetics Program, Department of Obstetrics and Gynecology, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada.

David Chitayat (D)

The Prenatal Diagnosis and Medical Genetics Program, Department of Obstetrics and Gynecology, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada.

Kelly L Gilmore (KL)

Division of Maternal Fetal Medicine, Department of Obstetrics and Gynecology, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.

Alexander A L Jorge (AAL)

Unidade de Endocrinologia Genética (LIM25) e Laboratório de Endocrinologia Celular e Molecular, Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo, Sao Paulo, Sao Paulo, Brazil.

Bruna L Freire (BL)

Unidade de Endocrinologia do Desenvolvimento, Laboratorio de Hormonios e Genetica Molecular (LIM42), Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo, Sao Paulo, São Paulo, Brazil.

Antonio Lerario (A)

Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes, University of Michigan, Ann Arbor, Michigan, USA.

Patrick Shannon (P)

Department of Pathology and Laboratory Medicine, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada.

Heidi Cope (H)

Center for Human Disease Modeling, Duke University, Durham, North Carolina, USA.

William B Gallentine (WB)

Department of Neurology and Pediatrics, Stanford University, Lucile Packard Children's Hospital, Palo Alto, California, USA.

Gwenal Le Guyader (G)

Service de génétique clinique, CHU de Poitiers, Poitiers, France.

Frederic Bilan (F)

Service de génétique clinique, CHU de Poitiers, Poitiers, France.
Laboratoire de Neurosciences Expérimentales et Cliniques, INSERM U1084, Université de Poitiers, Poitiers, France.

Pascaline Létard (P)

Service de génétique clinique, CHU de Poitiers, Poitiers, France.

Erica E Davis (EE)

Department of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA.
Department of Cell and Molecular Biology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA.
Stanley Manne Children's Research Institute, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

Neeta L Vora (NL)

Division of Maternal Fetal Medicine, Department of Obstetrics and Gynecology, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.

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Classifications MeSH