Author: Nguyen, Andrew PhD

6. ASOs Targeting the miR-29b Binding Site in the GRN mRNA Increase Translation of Progranulin: Potential Therapeutic Strategy for Progranulin-deficient Frontotemporal Dementia

Introduction: Frontotemporal dementia (FTD) is a devastating neurological disease with no approved treatment or cure. One common cause of FTD is heterozygous loss-of-function GRN mutations, which result in haploinsufficiency of progranulin. Antisense oligonucleotides (ASOs) are emerging as a promising therapeutic modality for neurological diseases, but ASO-based strategies for increasing target protein levels are limited. Several […]

7. Developing Antisense Oligonucleotides Targeting MiR-29b Binding Site to Increase Progranulin Protein Levels for Frontotemporal Dementia

Introduction: Frontotemporal dementia (FTD) is a devastating neurological disease with no approved treatment or cure. One common cause of FTD is heterozygous loss-of-function GRN mutations, which result in progranulin haploinsufficiency. Antisense oligonucleotides (ASOs) are emerging as a promising therapeutic modality for neurological diseases, but ASO-based strategies for increasing target protein levels are limited. Several microRNAs […]