DC8: Optimizing ASO brain uptake and retention time

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Supervisor: Dr. H. Tran

Host Institute: Institut de Recherches Servier, France (www.servier.com)

Secondments planned: Université de Versailles Saint-Quentin-en-Yvelines, France; Leiden University Medical Center, The Netherlands

Doctoral program: Doctoral program Life Sciences and Health of Université Paris-Saclay

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Project description:

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Antisense Oligonucleotide therapeutics are emerging as an innovative potentially transformative medicine for certain brain disorders with no therapeutic options such as spinocerebellar ataxias. We are interested in understanding better how sequences and/or modified chemistries can impact their potency, uptake, biodistribution, safety and elimination half-life with a particular focus on the cerebellum. The candidate will benefit from preliminary data generated in the host laboratory on protein modifier regulating ASO uptake. Biodistribution, potency and safety profile of a panel of different ASOs in cerebellum other brain regions in rodent and large animal species will also be leveraged to support the PhD thesis.