Article
Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation.
The Journal of biological chemistry - 1 Dec 2023
Aggarwal Geetika, Banerjee Subhashis, Jones Spencer A, Benchaar Yousri, Bélanger Jasmine, Sévigny Myriam, Smith Denise M, Niehoff Michael L, Pavlack Monica, de Vera Ian Mitchelle S, Petkau Terri L, Leavitt Blair R, Ling Karen, Jafar-Nejad Paymaan, Rigo Frank, Morley John E, Farr Susan A, Dutchak Paul A, Sephton Chantelle F, Nguyen Andrew D
Abstract excerpt
Heterozygous GRN (progranulin) mutations cause frontotemporal dementia (FTD) due to haploinsufficiency, and increasing progranulin levels is a major therapeutic goal. Several microRNAs, including miR-29b, negatively regulate progranulin protein levels. Antisense oligonucleotides (ASOs) are emerging as a promising therapeutic modality for neurological diseases, but strategies for increasing target protein levels...
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