Patient-Derived Cellular Models for Polytarget Precision Medicine in Pantothenate Kinase-Associated Neurodegeneration.
L-carnitine
fibroblasts
induced neurons
neurodegeneration with brain iron accumulation (NBIA)
omega 3
pantethine
pantothenate
pantothenate kinase 2 (PANK2)
pantothenate kinase-associated neurodegeneration (PKAN)
precision medicine
thiamine
vitamin E
α-lipoic acid
Journal
Pharmaceuticals (Basel, Switzerland)
ISSN: 1424-8247
Titre abrégé: Pharmaceuticals (Basel)
Pays: Switzerland
ID NLM: 101238453
Informations de publication
Date de publication:
26 Sep 2023
26 Sep 2023
Historique:
received:
04
09
2023
revised:
21
09
2023
accepted:
21
09
2023
medline:
28
10
2023
pubmed:
28
10
2023
entrez:
28
10
2023
Statut:
epublish
Résumé
The term neurodegeneration with brain iron accumulation (NBIA) brings together a broad set of progressive and disabling neurological genetic disorders in which iron is deposited preferentially in certain areas of the brain. Among NBIA disorders, the most frequent subtype is pantothenate kinase-associated neurodegeneration (PKAN) caused by pathologic variants in the
Identifiants
pubmed: 37895830
pii: ph16101359
doi: 10.3390/ph16101359
pmc: PMC10609847
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Subventions
Organisme : Instituto de Salud Carlos III
ID : PI19/00377 and PI22/00142 grants
Organisme : Junta de Andalucia
ID : PY18-850
Organisme : UPO-FEDER
ID : UPO-1380614
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