Professor of translational genetics
Prof. Dr. A. (Annemieke) Aartsma-Rus
Area(s) of expertise:
Duchenne muscular dystrophy, Antisense oligonucleotides
Duchenne muscular dystrophy, Antisense oligonucleotides
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Introduction
I am a professor of translational genetics at the Department of Human Genetics. I focus on optimizing antisense oligonucleotide (AON) therapy for Duchenne (also known as exon skipping). Furthermore, I aim to develop exon skipping for patients with very rare brain disorders within the Dutch Center of RNA Therapeutics.
I have an MSc in biomedical sciences and obtained my PhD from Leiden University in 2005 for research on exon skipping therapy development for Duchenne.
I have a visiting professorship in Newcastle University (John Walton Muscular Dystrophy Research Center).
I am a board member of the Dutch Center for RNA Therapeutics and Chair of the TREAT-NMD Advisory Committee for Therapeutics and the TREAT-NMD Educational Committee.
I am also on the advisory board of multiple (inter)national Duchenne organisations.
I have an MSc in biomedical sciences and obtained my PhD from Leiden University in 2005 for research on exon skipping therapy development for Duchenne.
I have a visiting professorship in Newcastle University (John Walton Muscular Dystrophy Research Center).
I am a board member of the Dutch Center for RNA Therapeutics and Chair of the TREAT-NMD Advisory Committee for Therapeutics and the TREAT-NMD Educational Committee.
I am also on the advisory board of multiple (inter)national Duchenne organisations.
Scientific research
I have received multiple awards (including the Duchenne Award from the Dutch Duchenne Parent Project in 2011, being selected as a member of De Jonge Akademie in 2014, the Ammodo Science Award in 2021 and the Black Pearl Science Award from Eurordis in 2021).
I have been the recipient of national and international grants from e.g. ZonMw (including a VIDI), Duchenne Parent Project, the Prinses Beatrix Spierfonds and for EU projects and networks.
The exon skipping therapy that I have helped pioneer and develop has been approved in the USA by the FDA based on IP from the LUMC.
My current research is focused on improving therapies for Duchenne muscular dystrophy and the development and characterization of models for this disease. In addition I aim to explore whether the therapeutic approach developed for Duchenne applies for patients with very rare brain diseases.
Ik heb verschillende prijzen mogen ontvangen (bv de Duchenne Award van het Nederlandse Duchenne Parent Project 2011, selectie als lid van De Jonge Akademie in 2014, Ammodo Prijs in 2021, Black Pearl Award Eurordis in 2021).
I have been the recipient of national and international grants from e.g. ZonMw (including a VIDI), Duchenne Parent Project, the Prinses Beatrix Spierfonds and for EU projects and networks.
The exon skipping therapy that I have helped pioneer and develop has been approved in the USA by the FDA based on IP from the LUMC.
My current research is focused on improving therapies for Duchenne muscular dystrophy and the development and characterization of models for this disease. In addition I aim to explore whether the therapeutic approach developed for Duchenne applies for patients with very rare brain diseases.
Ik heb verschillende prijzen mogen ontvangen (bv de Duchenne Award van het Nederlandse Duchenne Parent Project 2011, selectie als lid van De Jonge Akademie in 2014, Ammodo Prijs in 2021, Black Pearl Award Eurordis in 2021).
