Delivery of mRNA for the augmentation of proteins and enzymes in human genetic diseases

Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with prote...

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Bibliographic Details
Main Authors CONCINO, Michael, GUILD, Braydon Charles, HEARTLEIN, Michael, DeRosa, Frank
Format Patent
LanguageEnglish
Published 16.05.2024
Subjects
CHEMISTRY
DERIVATIVES THEREOF
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SUGARS
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Summary:Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.
Bibliography:Application Number: AU20200202755