

DC5 - Pilar Maestre
My deepest motivation has always been the belief that true fulfilment comes from contributing to the well-being of others. I see this as the ultimate mission of any meaningful career, and for me, research provides the perfect way to unite this purpose with my fascination for nature and science.
With this in mind, I pursued a bachelor’s degree in Biotechnology in my hometown, Valencia, followed by a master’s degree in Translational Medical Research at the University of Heidelberg, Germany, as I was intrigued by the process of taking discoveries from bench to bedside. For my master’s thesis, I worked at the Institute of Bioengineering of Catalonia, where I developed a drug-screening platform targeting sarcolemma damage in Duchenne muscular dystrophy using an optogenetic system and 3D disease models.
I am now beginning a new chapter of my career as a Marie Curie PhD fellow within the EFFecT consortium, under the supervision of Aurélie Goyenvalle at Université de Versailles Saint-Quentin-en-Yvelines (UVSQ), Versailles, France. My doctoral project focuses on the characterization of ASO-intracellular protein partners to increase the therapeutic index of antisense oligonucleotides, with the aim of advancing the efficacy of RNA-based therapies for neuromuscular diseases.
Outside of the lab, I enjoy traveling and exploring new cultures, particularly through their cuisine. I am also passionate about outdoor sports and recently took up surfing, although my main priority now is to learn French.
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DC5: Characterization of ASO-intracellular protein partners to increase ASO therapeutic index
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Supervisor: Dr. A. Goyenvalle
External mentor: Prof. L. Desviat
Host Institute: Université de Versailles Saint-Quentin-en-Yvelines, France (www.uvsq.fr)
Secondments planned: Radboud University Medical Center, The Netherlands; Leiden University Medical Center, The Netherlands
Doctoral program: Doctoral program Life Sciences and Health of Université Paris-Saclay
Starting date: October 30th, 2025
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Project description:
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This project aims at studying one of the main bottlenecks of ASO-based therapeutics by identifying ASO-protein partners which could significantly increase ASO potency. In this context and following a proteomic and functional screen already performed in the host laboratory, we propose to determine and subsequently validate the proteins promoting and inhibiting the ASO uptake and trafficking to their RNA-targets. Considering the expertise of the host laboratory in neuromuscular diseases, DC5 will be working on exon skipping therapies for Duchenne muscular dystrophy, using both in vitro and in vivo models of the disease. Identified proteins/genes will be specifically downregulated in vitro in muscle cells and the most promising candidates impacting the effect of ASO will be evaluated in vivo in DMD mouse models.