ESR Project 1
Astra Zeneca AB, Discovery Sciences, Gothenburg, Sweden
Enhancing ASO action and mRNA regulation in cardio metabolic disease
Lille University, France 2013 - 2019
Lille University, France 2016 & 2019
The aim of this ESR PhD project is the evaluation of different alternatives for enhancement of mRNA transcription:
RNA activation that affects the transcription of mRNA as well as targeting of miRNAs that suppress mRNA transcription, the latter in collaboration with KI.
ON constructs for enhancement of mRNA transcription, processing and translation. This involves ONs that affect splice switching and activation of transcription by ONs targeting microRNAs that suppress transcription in collaboration with KI and SLL.
ONs conjugated to multiple entities for increased efficacy of ON therapy, including entities for increased tissue specificity and cell uptake, will be evaluated in vitro in relevant cell types especially for protein replacement therapy for cardiometabolic disease.
During secondments in partner organizations Quentin will extend his knowledge on the effect of ONs derivatization and modification on RNA transcription or translation.
This project is carried out in strong collaboration with the following groups:
Prof. Roger Strömberg, Karolinska Institutet, Sweden
Prof. Edvard Smith, SLL, Sweden
Dr. Sean Lim, Prof. Alex Mirnezami, Southampton Cancer Centre (UHSFT), Southampton, UK
Project summary in 3 min
We have state-of-the-art purpose-built laboratories with facilities to develop small molecules and nucleotide therapeutics by means of molecular and chemical biology approaches. This includes a wide range of facilities from target identification, compound design and selection; compound synthesis, purification and analysis; compound library generation; high-throughput assays for identifying active compounds; cell and animal model-based preclinical studies; bioinformatics and biomarker discovery; drug delivery as well as clinical trials of drug compounds ( small molecules and oligonucleotides). Expertise in commercialisation (invention-to-market process) and IP protection.