Search Results - "Evers, Melvin M."

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model by 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

“…Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the huntingtin gene. Previously, we showed…”
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Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy by 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

“…A hexanucleotide GGGGCC expansion in intron 1 of chromosome 9 open reading frame 72 (C9orf72) gene is the most frequent cause of amyotrophic lateral sclerosis…”
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Human Brain Organoids as Models for Central Nervous System Viral Infection by Depla, Josse A, Mulder, Lance A, de Sá, Renata Vieira, Wartel, Morgane, Sridhar, Adithya, Evers, Melvin M, Wolthers, Katja C, Pajkrt, Dasja

“…Pathogenesis of viral infections of the central nervous system (CNS) is poorly understood, and this is partly due to the limitations of currently used…”
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A Perspective on Organoids for Virology Research by Sridhar, Adithya, Simmini, Salvatore, Ribeiro, Carla M S, Tapparel, Caroline, Evers, Melvin M, Pajkrt, Dasja, Wolthers, Katja

“…Animal models and cell lines are invaluable for virology research and host-pathogen interaction studies. However, it is increasingly evident that these models…”
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Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients by 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

“…The most common pathogenic mutation in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) is an intronic GGGGCC (G4C2) repeat in the…”
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AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models by Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.

“…Huntington disease (HD) is a fatal neurodegenerative disorder caused by an autosomal dominant CAG repeat expansion in the huntingtin (HTT) gene. The translated…”
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AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes by Keskin, Sonay, Brouwers, Cynthia C., Sogorb-Gonzalez, Marina, Martier, Raygene, Depla, Josse A., Vallès, Astrid, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.

“…Huntington disease (HD) is a fatal neurodegenerative genetic disorder, thought to reflect a toxic gain of function in huntingtin (Htt) protein…”
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Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon by 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

“…Abstract Spinocerebellar ataxia type 3 is caused by a polyglutamine expansion in the ataxin-3 protein, resulting in gain of toxic function of the mutant…”
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Targeting several CAG expansion diseases by a single antisense oligonucleotide by 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

“…To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a…”
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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 by 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

“…Spinocerebellar ataxia type 3 (SCA3) is a progressive neurodegenerative disorder caused by expansion of the polyglutamine repeat in the ataxin-3 protein…”
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Enterovirus D68 Infection in Human Primary Airway and Brain Organoids: No Additional Role for Heparan Sulfate Binding for Neurotropism by 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

“…Enterovirus D68 (EV-D68) is an RNA virus that can cause outbreaks of acute flaccid paralysis (AFP), a polio-like disease. Before 2010, EV-D68 was a rare…”
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Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease by 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.

“…Spinocerebellar ataxia type 3 (SCA3), or Machado-Joseph disease (MJD), is a progressive neurodegenerative disorder caused by a CAG expansion in the ATXN3 gene…”
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Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain by 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.

“…The development of gene therapies for central nervous system disorders is challenging because it is difficult to translate preclinical data from current…”
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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 by Spronck, Elisabeth A, Vallès, Astrid, Lampen, Margit H, Montenegro-Miranda, Paula S, Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M, Petry, Harald, Deventer, Sander J van, Konstantinova, Pavlina, Haan, Martin de

“…Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key…”
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Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic by Miniarikova, Jana, Evers, Melvin M., Konstantinova, Pavlina

“…The single mutation underlying the fatal neuropathology of Huntington’s disease (HD) is a CAG triplet expansion in exon 1 of the huntingtin (HTT) gene, which…”
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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 by 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

“…Abstract Huntington disease (HD) is a fatal neurodegenerative disease caused by a pathogenic expansion of a CAG repeat in the huntingtin (HTT) gene. There are…”
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AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington’s disease mouse model by 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

“…Abstract Huntingtin (HTT)-lowering therapies show great promise in treating Huntington’s disease. We have developed a microRNA targeting human HTT that is…”
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Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain by Zalachoras, Ioannis, Evers, Melvin M, van Roon-Mom, Willeke M C, Aartsma-Rus, Annemieke M, Meijer, Onno C

“…A limiting factor in brain research still is the difficulty to evaluate in vivo the role of the increasing number of proteins implicated in neuronal processes…”
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Antisense oligonucleotides in therapy for neurodegenerative disorders by Evers, Melvin M., Toonen, Lodewijk J.A., van Roon-Mom, Willeke M.C.

“…Antisense oligonucleotides are synthetic single stranded strings of nucleic acids that bind to RNA and thereby alter or reduce expression of the target RNA…”
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Emerging Therapies for Huntington’s Disease – Focus on N-Terminal Huntingtin and Huntingtin Exon 1 by van der Bent, M. Leontien, Evers, Melvin M, Valles, Astrid

“…Huntington's disease is a devastating heritable neurodegenerative disorder that is caused by the presence of a trinucleotide CAG repeat expansion in the…”
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