LIPID NANOPARTICLES CONTAINING POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE AND USES THEREOF

This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and/or activity in subjects and are useful f...

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Bibliographic Details
Main Authors Cornebise, Mark, Hennessy, Edward, Benenato, Kerry
Format Patent
LanguageEnglish
Published 27.06.2024
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary:This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and/or activity in subjects and are useful for the treatment of glycogen storage disease type 1a (GSD-Ia). Lipid nanoparticle/mRNA therapies of the invention increase glucose production and reduce the abnormal accumulation of glycogen and glucose-6-phosphate associated with GSD-Ia.
Bibliography:Application Number: US202218282643