Raygene M. Martier
Leiden, ZH, Netherlands
Last Updated: 09/26/2026
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Found 10 publications
Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock.
Journal: Frontiers in neuroscience
Published: July 05, 2020
Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery.
Journal: Gene therapy
Published: November 22, 2019
Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease.
Journal: Molecular therapy. Methods & clinical development
Published: July 11, 2019
AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes.
Journal: Molecular therapy. Methods & clinical development
Published: March 03, 2019
Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy.
Journal: Molecular therapy. Nucleic acids
Published: December 10, 2018
In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver.
Journal: Molecular therapy. Methods & clinical development
Published: December 10, 2018
Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients.
Journal: Molecular therapy. Nucleic acids
Published: September 19, 2018
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
Journal: Gene therapy
Published: January 19, 2017
Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.
Journal: Molecular therapy. Nucleic acids
Published: September 15, 2015
Showing 1-10 of 10
Last Updated: 09/26/2026