Leading scientists from UKT intensively investigate and pursue clinical translation of key Oligonucleotide Therapy approaches:
- Gapmers and splice modulating antisense-oligonucleotides (ASOs) for a large set of neurodegenerative diseases such as spinocerebellar ataxias (SCAs), complex autosomal-recessive ataxias (ARCAS), genetic forms of Parkinson’s disease, hereditary spastic paraplegias (HSPs), and neurodegeneration with brain iron accumulation (NBIAs);
- siRNAs for a wide range of immunological and hematological diseases, including Graft-versus-Host Disease, congenital neutropenia, and RNA-modified cellular immunotherapies.
- RNA base editing ASOs for liver, CNS and eye diseases.
PIs:
