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RNA interference-mediated therapy

Frontotemporal dementianeurodegenerative diseasesRare diseasesShort interfering RNAsTherapy

Introduction

The present invention concerns an RNA interference mediated therapy for neurodegenerative diseases associated with abnormalities of microtubule-associated protein tau (“tauopathies”).

Technical features

The invention consists in the application of RNA interference to specifically degrade MAPT mRNA containing exon 10. The altered inclusion of exon 10 in MAPT transcript, which codes for protein tau, is the cause of Frontotemporal Dementia with Parkinsonism linked to chromosome 17 (FTDP-17). No cures are presently available for FTDP-17 or other tauopathies.

Possible Applications

  • Therapy for neurodegenerative diseases;
  • Therapeutic agent for the cure of Frontotemporal Dementia with Parkinsonism linked to Chromosome 17;
  • Therapeutic agent effective in the treatment of neurodegenerative diseases associated with abnormalities of MAPT gene encoded protein tau.

Advantages

  • Tissue specificity of the effect, dictated by the fact that siRNAs are only acting in those tissues where the aberrant splicing isoform is present;
  • Target specificity (reduced off-target effects) due to the mechanism of action based on nucleic acids hybridisation.