QRX-704
/ ProQR
- LARVOL DELTA
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August 10, 2022
A pathogenic proteolysis-resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function.
(PubMed, JCI Insight)
- "Furthermore, QRX-704 treatments resulted in a reduction of HTT aggregation and an increase in dendritic spine count. Thus, ASO-induced HTT exon12 splicing-switching from HTT may provide a novel therapeutic strategy for HD."
Journal • CNS Disorders • Huntington's Disease • Movement Disorders • Targeted Protein Degradation • CASP6
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