Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model-Reference-Cited by-同舟云学术

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Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model

Published:2022-09 Issue: Volume:32 Page:100882
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Diaz-Trelles Ramon,Lee Sharon,Kuakini Kristen,Park Jenny,Dukanovic Adrian,Gonzalez Jose A.,Dam Thanhchau,Kim Jae Heon,Vega Jerel Boyd,Sablad Marciano,Karmali Priya P.,Tachikawa Kiyoshi,Chivukula Padmanabh

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference11 articles.

1. Phenylketonuria: phenylalanine neurotoxicity;Giżewska,2015

2. Phenylketonuria;Blau;Lancet,2010

3. Evaluation of nutritional status and pathophysiology of growth retardation in patients with phenylketonuria;Dobbelaere;J. Inherit. Metab. Dis.,2003

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2. Unleashing the potential of mRNA therapeutics for inherited neurological diseases;Brain;2024-04-25

3. Inborn errors of amino acid metabolism – from underlying pathophysiology to therapeutic advances;Disease Models & Mechanisms;2023-11-01

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