Current treatment for citrin deficiency during NICCD and adaptation/compensation stages: Strategy to prevent CTLN2.


Journal

Molecular genetics and metabolism
ISSN: 1096-7206
Titre abrégé: Mol Genet Metab
Pays: United States
ID NLM: 9805456

Informations de publication

Date de publication:
07 2019
Historique:
received: 02 05 2019
revised: 14 06 2019
accepted: 14 06 2019
pubmed: 1 7 2019
medline: 10 4 2020
entrez: 1 7 2019
Statut: ppublish

Résumé

Identification of the genes responsible for adult-onset type II citrullinemia (CTLN2) and citrin protein function have enhanced our understanding of citrin deficiency. Citrin deficiency is characterized by 1) neonatal intrahepatic cholestasis caused by citrin deficiency (NICCD); 2) adaptation/compensation stage with unique food preference from childhood to adulthood; and 3) CTLN2. The treatment of NICCD aims to prevent the progression of cholestasis, and it includes medium chain triglycerides (MCT) milk and lactose-free milk, in addition to medications (e.g., vitamin K

Identifiants

pubmed: 31255436
pii: S1096-7192(19)30327-0
doi: 10.1016/j.ymgme.2019.06.004
pii:
doi:

Substances chimiques

Calcium-Binding Proteins 0
Mitochondrial Membrane Transport Proteins 0
Organic Anion Transporters 0
Triglycerides 0
Vitamins 0
citrin 1340-08-5

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

175-183

Informations de copyright

Copyright © 2019 The Authors. Published by Elsevier Inc. All rights reserved.

Auteurs

Yoshiyuki Okano (Y)

Okano Children's Clinic, and Department of Pediatrics, Hyogo College of Medicine, 1-20-1 Izumifutyu, Izumi 594-0071, Japan. Electronic address: okano@med.osaka-cu.ac.jp.

Toshihiro Ohura (T)

Division of Pediatrics, Sendai City Hospital, 1-1-1 Asutonagamachi, Taihaku-ku, Sendai 982-8502, Japan; Department of Pediatrics, Tohoku University School of Medicine, 1-1 Seiryomachi, Aobaku, Sendai 980-8574, Japan.

Osamu Sakamoto (O)

Department of Pediatrics, Tohoku University School of Medicine, 1-1 Seiryomachi, Aobaku, Sendai 980-8574, Japan.

Ayano Inui (A)

Department of Pediatric Hepatology and Gastroenterology, Saiseikai Yokohamashi Tobu Hospital, Kanagawa, Japan.

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Classifications MeSH