Novel variant in HPS3 gene in a patient with Hermansky Pudlak syndrome (HPS) type 3.


Journal

Platelets
ISSN: 1369-1635
Titre abrégé: Platelets
Pays: England
ID NLM: 9208117

Informations de publication

Date de publication:
02 Oct 2020
Historique:
pubmed: 28 12 2019
medline: 29 6 2021
entrez: 28 12 2019
Statut: ppublish

Résumé

Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive disorder caused by defects in 10 human sequencing revealed a novel pathogenic homozygous variant (NM_032383.4:c.7>T, p.Gln3*) resulting in a premature stop codon at the amino acid 3. Moreover, our report highlights the importance of evaluating platelet function in children with OCA without bleeding diathesis to identify HPS early and prevent bleeding complications.

Identifiants

pubmed: 31880485
doi: 10.1080/09537104.2019.1704716
doi:

Substances chimiques

HPS3 protein, human 0
Intracellular Signaling Peptides and Proteins 0

Types de publication

Case Reports Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

960-963

Auteurs

Anna Lecchi (A)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center , Milano, Italy.

Silvia La Marca (S)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center , Milano, Italy.

Eti A Femia (EA)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center , Milano, Italy.

Antonia Lenz (A)

Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Faculty of Medicine, Medical Center - University of Freiburg , Germany.

Doris Boeckelmann (D)

Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Faculty of Medicine, Medical Center - University of Freiburg , Germany.

Andrea Artoni (A)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center , Milano, Italy.

Flora Peyvandi (F)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center , Milano, Italy.
Department of Pathophysiology and Transplantation and Fondazione Luigi Villa, Università degli Studi di Milano , Milano, Italy.

Barbara Zieger (B)

Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Faculty of Medicine, Medical Center - University of Freiburg , Germany.

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Classifications MeSH