AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model von Evers, Melvin M., Miniarikova, Jana, Juhas, Stefan, Vallès, Astrid, Bohuslavova, Bozena, Juhasova, Jana, Skalnikova, Helena Kupcova, Vodicka, Petr, Valekova, Ivona, Brouwers, Cynthia, Blits, Bas, Lubelski, Jacek, Kovarova, Hana, Ellederova, Zdenka, van Deventer, Sander J., Petry, Harald, Motlik, Jan, Konstantinova, Pavlina

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Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy von Martier, Raygene, Liefhebber, Jolanda M., García-Osta, Ana, Miniarikova, Jana, Cuadrado-Tejedor, Mar, Espelosin, Maria, Ursua, Susana, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina

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Human Brain Organoids as Models for Central Nervous System Viral Infection von Depla, Josse A, Mulder, Lance A, de Sá, Renata Vieira, Wartel, Morgane, Sridhar, Adithya, Evers, Melvin M, Wolthers, Katja C, Pajkrt, Dasja

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A Perspective on Organoids for Virology Research von Sridhar, Adithya, Simmini, Salvatore, Ribeiro, Carla M. S., Tapparel, Caroline, Evers, Melvin M., Pajkrt, Dasja, Wolthers, Katja

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Targeting several CAG expansion diseases by a single antisense oligonucleotide von Evers, Melvin M, Pepers, Barry A, van Deutekom, Judith C T, Mulders, Susan A M, den Dunnen, Johan T, Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B, van Roon-Mom, Willeke M C

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Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients von Martier, Raygene, Liefhebber, Jolanda M., Miniarikova, Jana, van der Zon, Tom, Snapper, Jolanda, Kolder, Iris, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina

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Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon von Evers, Melvin M, Tran, Hoang-Dai, Zalachoras, Ioannis, Pepers, Barry A, Meijer, Onno C, den Dunnen, Johan T, van Ommen, Gert-Jan B, Aartsma-Rus, Annemieke, van Roon-Mom, Willeke M.C

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AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models von Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.

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AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes von Keskin, Sonay, Brouwers, Cynthia C., Sogorb-Gonzalez, Marina, Martier, Raygene, Depla, Josse A., Vallès, Astrid, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.

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Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model von Toonen, Lodewijk J A, Overzier, Maurice, Evers, Melvin M, Leon, Leticia G, van der Zeeuw, Sander A J, Mei, Hailiang, Kielbasa, Szymon M, Goeman, Jelle J, Hettne, Kristina M, Magnusson, Olafur Th, Poirel, Marion, Seyer, Alexandre, 't Hoen, Peter A C, van Roon-Mom, Willeke M C

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Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic von Miniarikova, Jana, Evers, Melvin M., Konstantinova, Pavlina

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Enterovirus D68 Infection in Human Primary Airway and Brain Organoids: No Additional Role for Heparan Sulfate Binding for Neurotropism von Sridhar, Adithya, Depla, Josse A, Mulder, Lance A, Karelehto, Eveliina, Brouwer, Lieke, Kruiswijk, Leonie, Vieira de Sá, Renata, Meijer, Adam, Evers, Melvin M, van Kuppeveld, Frank J M, Pajkrt, Dasja, Wolthers, Katja C

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Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain von Depla, Josse A., Sogorb-Gonzalez, Marina, Mulder, Lance A., Heine, Vivi M., Konstantinova, Pavlina, van Deventer, Sander J., Wolthers, Katja C., Pajkrt, Dasja, Sridhar, Adithya, Evers, Melvin M.

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Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease von Martier, Raygene, Sogorb-Gonzalez, Marina, Stricker-Shaver, Janice, Hübener-Schmid, Jeannette, Keskin, Sonay, Klima, Jiri, Toonen, Lodewijk J., Juhas, Stefan, Juhasova, Jana, Ellederova, Zdenka, Motlik, Jan, Haas, Eva, van Deventer, Sander, Konstantinova, Pavlina, Nguyen, Huu Phuc, Evers, Melvin M.

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Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology von Spronck, Elisabeth A., Valles, Astrid, Lampen, Margit H., Montenegro-Miranda, Paula S., Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M., Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, de Haan, Martin

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Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease von Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R

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AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington’s disease mouse model von Thomson, Sarah B, Stam, Anouk, Brouwers, Cynthia, Fodale, Valentina, Bresciani, Alberto, Vermeulen, Michael, Mostafavi, Sara, Petkau, Terri L, Hill, Austin, Yung, Andrew, Russell-Schulz, Bretta, Kozlowski, Piotr, MacKay, Alex, Ma, Da, Beg, Mirza Faisal, Evers, Melvin M, Vallès, Astrid, Leavitt, Blair R

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Emerging Therapies for Huntington’s Disease – Focus on N-Terminal Huntingtin and Huntingtin Exon 1 von van der Bent, M. Leontien, Evers, Melvin M, Valles, Astrid

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Antisense oligonucleotides in therapy for neurodegenerative disorders von Evers, Melvin M., Toonen, Lodewijk J.A., van Roon-Mom, Willeke M.C.

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miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3 von Nobre, Rui Jorge, Lobo, Diana D, Henriques, Carina, Duarte, Sonia P, Lopes, Sara M, Silva, Ana C, Lopes, Miguel M, Mariet, Fanny, Schwarz, Lukas K, Baatje, M S, Ferreira, Valerie, Vallès, Astrid, Pereira de Almeida, Luis, Evers, Melvin M, Toonen, Lodewijk J A

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