Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
AssigneesPresident and Fellows of Harvard College, Children's Medical Center Corporation
InventorsPeter Jungsoo Park, Jin Kuk Kim, Timothy Yu, Yu-Han Huang
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Abstract
Described herein are methods and compositions related to the modulation of progranulin expression or activity in the brain for the treatment of neurodegenerative diseases.
Claims11
An antisense oligonucleotide (ASO) comprising an oligonucleotide of 20 or more linked nucleotides or modified nucleotides in length, wherein the oligonucleotide comprises a nucleobase sequence targeting a Progranulin (GRN) mRNA to increase GRN expression or activity, wherein the oligonucleotide has at least 80% sequence identity to the nucleotide sequence of SEQ ID NO:
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Dependencies are inferred from the claim text.