Next-Generation Sequencing Reveals A JAGN1 Mutation in a Syndromic Child With Intermittent Neutropenia.


Journal

Journal of pediatric hematology/oncology
ISSN: 1536-3678
Titre abrégé: J Pediatr Hematol Oncol
Pays: United States
ID NLM: 9505928

Informations de publication

Date de publication:
05 2019
Historique:
pubmed: 26 7 2018
medline: 18 12 2019
entrez: 26 7 2018
Statut: ppublish

Résumé

Jagunal homolog 1 (JAGN1) gene was identified as a novel responsible for severe congenital neutropenia. The protein encoded by this gene is required for neutrophil differentiation, survival and function in microbial activity. JAGN1-deficient human neutrophils are characterized by alterations in trafficking within the endoplasmic reticulum and golgi compartments because of ultrastructural defects in endoplasmic reticulum and susceptibility to apoptosis. We report a patient exhibiting an intermittent neutropenia, for which a next-generation sequencing revealed a homozygous mutation in the JAGN1 gene. The patient extends the clinical variability associated to JAGN1 mutations, and this case highlights the importance of genetic investigations in patients with suspected neutropenia.

Sections du résumé

BACKGROUND
Jagunal homolog 1 (JAGN1) gene was identified as a novel responsible for severe congenital neutropenia. The protein encoded by this gene is required for neutrophil differentiation, survival and function in microbial activity. JAGN1-deficient human neutrophils are characterized by alterations in trafficking within the endoplasmic reticulum and golgi compartments because of ultrastructural defects in endoplasmic reticulum and susceptibility to apoptosis.
OBSERVATIONS
We report a patient exhibiting an intermittent neutropenia, for which a next-generation sequencing revealed a homozygous mutation in the JAGN1 gene.
CONCLUSIONS
The patient extends the clinical variability associated to JAGN1 mutations, and this case highlights the importance of genetic investigations in patients with suspected neutropenia.

Identifiants

pubmed: 30044346
doi: 10.1097/MPH.0000000000001256
doi:

Substances chimiques

JAGN1 protein, human 0
Membrane Proteins 0

Types de publication

Case Reports Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

e266-e269

Auteurs

Cristina Cifaldi (C)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.

Jessica Serafinelli (J)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.

Davide Petricone (D)

Department of Systems Medicine, "University of Rome Tor Vergata," Rome.

Immacolata Brigida (I)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute.

Silvia Di Cesare (S)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.

Gigliola Di Matteo (G)

Department of Systems Medicine, "University of Rome Tor Vergata," Rome.

Maria Chiriaco (M)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.

Rita De Vito (R)

Department of Pathology and Molecular Histopathology, Bambino Gesù Children's Hospital IRCCS.

Giuseppe Palumbo (G)

University Department of Pediatrics, Unit of Hematology and Oncology, Bambino Gesù Children's Hospital.

Paolo Rossi (P)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.
Department of Systems Medicine, "University of Rome Tor Vergata," Rome.

Paolo Palma (P)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.

Caterina Cancrini (C)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.
Department of Systems Medicine, "University of Rome Tor Vergata," Rome.

Alessandro Aiuti (A)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute.
Vita Salute San Raffaele University.
Pediatric Immunohematology, San Raffaele Scientific Institute, Milan, Italy.

Andrea Finocchi (A)

University Department of Pediatrics, Unit of Immune and Infectious Diseases, Childrens' Hospital Bambino Gesù.
Department of Systems Medicine, "University of Rome Tor Vergata," Rome.

Articles similaires

Genome, Chloroplast Phylogeny Genetic Markers Base Composition High-Throughput Nucleotide Sequencing

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C

Classifications MeSH