RNA Therapeutics Lab
RNA therapeutics are a broad class of RNA-based or RNA-targeting molecules spanning from short antisense oligonucleotides (ONs) to long modified messenger RNA or CRISPR/Cas9 systems. These modalities can be used to manipulate gene expression for therapeutic purposes, for example to induce gene silencing/activation, modulate splicing or direct gene editing. Our research group focuses on the development of aforementioned RNA therapeutics and application in various neurodegenerative, neuromuscular and inflammatory disease models. This line of research is accompanied with development of effective and safe drug delivery technologies to enhance the bioavailability of RNA therapeutics with a goal of facilitating their translation into clinics.
The main lines of our research are:
1. Development of novel antisense oligonucleotide-based compounds for splice switching applications.
2. Development of drug delivery systems and formulation technologies for mRNA- and CRISPR/Cas9-based systems.
3. Programming of drug delivery systems for targeted delivery.
4. Studying the mechanism of cellular uptake of therapeutic oligonucleotides and drug delivery systems.
Interested in joining us, please contact the PI directly (firstname.lastname@example.org)
Helena Sork (email@example.com)
Tõnis Lehto (firstname.lastname@example.org)