Marina Sogorb-González

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

Sonay Keskin, Cynthia Brouwers, Marina Sogorb-González et al. · Molecular Therapy — Methods & Clinical Development · 2019 · 70 citations · Full text

Life Sciences, Molecular Biology, Neurobiology +17

Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease

Raygene Martier, Marina Sogorb-González, Janice Stricker-Shaver et al. · Molecular Therapy — Methods & Clinical Development · 2019 · 58 citations · Full text

Life Sciences, Genetics, Neuroscience +17

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

Josse A. Depla, Marina Sogorb-González, Lance Mulder et al. · Molecular Therapy — Methods & Clinical Development · 2020 · 51 citations · Full text

Pharmacy, Biotechnology, Neuroscience +16

Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

Astrid Vallès, Melvin M. Evers, Anouk Stam et al. · Science Translational Medicine · 2021 · 51 citations

Life Sciences, Neurology, Neuroscience +17

Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models

Marina Sogorb-González, Christian Landles, Nicholas S. Caron et al. · Brain · 2024 · 21 citations · Full text

Life Sciences, Genetics, Molecular Biology +17