Annemieke Aartsma-Rus

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Professor of Translational Genetics

Leiden University Medical Center

Netherlands

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Professor Annemieke Aartsma-Rus is a faculty member at Leiden University Medical Center in the Netherlands. Her research focuses on translational genetics, particularly in the areas of antisense RNA therapies and individualized nucleic acid treatments for muscular dystrophies and neurodevelopmental disorders. She has authored several recent articles addressing the clinical development of antisense oligonucleotides and collaborative efforts in RNA therapeutics.

Recent Grants

Grant: Close

SCA7 as a showcase for personalized RNA therapy development in the Netherlands

Open Date: 2022-01-01

Close Date: 2024-12-01

Grant: Open

hiPS derived neurons for research and treatment of neurodevelopmental disorders

Open Date: 2022-01-01

Close Date: 2026-12-01

Grant: Close

Development of accurate gene repair therapy for Duchenne muscular dystrophy

Open Date: 2022-01-01

Close Date: 2024-01-01

Grant: Close

Ammodo: studying dystrophin transcript processing in detail

Open Date: 2021-11-01

Close Date: 2024-05-01

Grant: Close

Developing lipid nanoparticles for muscle specific delivery of genetic therapy tools

Open Date: 2021-01-01

Close Date: 2022-01-01

Articles (19)

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2023

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2023

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2023

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2022

Collaborators (12)

Willeke van Roon-Mom

Assistant Professor

Leiden University Medical Center

NETHERLANDS

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Jonathan K. Watts

University of Massachusetts Chan Medical School

UNITED STATES

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Jerry Vockley

University of Pittsburgh

UNITED STATES

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R Rodney Howell

Professor and Chair, then Emeritus 2019

University of Miami School of Medicine

UNITED STATES

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Laura Sepp-Lorenzino

Chief Scientific Officer

UNITED STATES

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Alex Garanto

Assistant Professor & Junior Principal Investigator

Radboudumc

NETHERLANDS

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Pietro Spitali

Assistant Professor

Universitair Medisch Centrum Utrecht

NETHERLANDS

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Matthis Synofzik

Professor

Hertie Institute for clinical brain research

GERMANY

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Mirko Signorelli

Assistant professor

Leiden University

NETHERLANDS

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Jennifer Cohen

Assistant Professor of Pediatrics, Division of Medical Genetics

Duke University

UNITED STATES

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Tamim Abdelaal

Delft University of Technology

NETHERLANDS

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A. Mahfouz

NETHERLANDS

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Annemieke Aartsma-Rus

Research Interests

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About

Recent Grants

Articles (19)

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

Antisense RNA therapies for muscular dystrophies

A historical perspective on the development of antisense oligonucleotide treatments for Duchenne muscular dystrophy and spinal muscular atrophy

Advancing ASO therapies from development to implementation

Treatability of the KMT2-Associated Neurodevelopmental Disorders Using Antisense Oligonucleotide-Based Treatments

Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments

A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides

Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics

Whole‐genome sequencing holds the key to the success of gene‐targeted therapies

Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations

From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis

Multiomic characterization of disease progression in mice lacking dystrophin

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon

Spatial transcriptomics reveal markers of histopathological changes in Duchenne muscular dystrophy mouse models

The Future of Exon Skipping for Duchenne Muscular Dystrophy

Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy

Next steps for the optimization of exon therapy for Duchenne muscular dystrophy

Applying Lessons Learned from Developing Exon Skipping for Duchenne to Developing Individualized Exon Skipping Therapy for Patients with Neurodegenerative Diseases

Consensus Guidelines for the Design and <i>In Vitro</i> Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides.

Collaborators (12)

Willeke van Roon-Mom

Jonathan K. Watts

Jerry Vockley

R Rodney Howell

Laura Sepp-Lorenzino

Alex Garanto

Pietro Spitali

Matthis Synofzik

Mirko Signorelli

Jennifer Cohen

Tamim Abdelaal

A. Mahfouz

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