Development of an mRNA replacement therapy for phenylketonuria.
LNP
MT: Delivery Strategies
PAH
PKU
hepatocytes
lipid nanoparticle
liver
mRNA
phenylketonuria
replacement
therapy
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
14 Jun 2022
14 Jun 2022
Historique:
received:
03
01
2022
accepted:
25
02
2022
entrez:
31
3
2022
pubmed:
1
4
2022
medline:
1
4
2022
Statut:
epublish
Résumé
Phenylketonuria (PKU) is an inborn error caused by deficiencies in phenylalanine (Phe) metabolism. Mutations in the phenylalanine hydroxylase (PAH) gene are the main cause of the disease whose signature hallmarks of toxically elevated levels of Phe accumulation in plasma and organs such as the brain, result in irreversible intellectual disability. Here, we present a unique approach to treating PKU deficiency by using an mRNA replacement therapy. A full-length mRNA encoding human PAH (hPAH) is encapsulated in our proprietary lipid nanoparticle LUNAR and delivered to a
Identifiants
pubmed: 35356682
doi: 10.1016/j.omtn.2022.02.020
pii: S2162-2531(22)00048-8
pmc: PMC8933640
doi:
Types de publication
Journal Article
Langues
eng
Pagination
87-98Informations de copyright
© 2022 The Authors.
Déclaration de conflit d'intérêts
The work included in this manuscript was performed using funds provided by Arcturus Therapeutics, Inc. The authors are current or former employees of Arcturus Therapeutics Inc.
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