M
Melvin M. Evers
Researcher at Leiden University Medical Center
Publications - 37
Citations - 1268
Melvin M. Evers is an academic researcher from Leiden University Medical Center. The author has contributed to research in topics: Huntingtin & Huntingtin Protein. The author has an hindex of 17, co-authored 32 publications receiving 904 citations.
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Journal ArticleDOI
Antisense oligonucleotides in therapy for neurodegenerative disorders.
TL;DR: There has been a recent revival of interest in the use of antisense oligonucleotides to treat several neurodegenerative disorders using different approaches to prevent disease onset or halt disease progression and the first clinical trials for spinal muscular atrophy and amyotrophic lateral sclerosis showing promising results.
Journal ArticleDOI
AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
Melvin M. Evers,Jana Miniarikova,Stefan Juhas,Astrid Vallès,Bozena Bohuslavova,Jana Juhasova,Helena Kupcova Skalnikova,Petr Vodicka,Ivona Valekova,Cynthia Brouwers,Bas Blits,Jacek Lubelski,Hana Kovarova,Zdenka Ellederova,Sander J. H. van Deventer,Harald Petry,Jan Motlik,Pavlina Konstantinova +17 more
TL;DR: The combination of widespread vector distribution and extensive huntingtin lowering observed with AAV5-miHTT supports the translation of a huntingtin-lowering gene therapy for HD from preclinical studies into the clinic.
Journal ArticleDOI
Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide
Melvin M. Evers,Barry A. Pepers,Judith C.T. van Deutekom,Susan A. M. Mulders,Johan T. den Dunnen,Annemieke Aartsma-Rus,Gert-Jan B. van Ommen,Willeke M. C. van Roon-Mom +7 more
TL;DR: This antisense oligonucleotide is not only a promising therapeutic tool to reduce mutant huntingtin levels in Huntington's disease but the results in spinocerebellar ataxia and dentatorubral-pallidoluysian atrophy cells suggest that this could also be applicable to other polyglutamine expansion disorders as well.
Journal ArticleDOI
Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon
Melvin M. Evers,Hoang-Dai Tran,Ioannis Zalachoras,Barry A. Pepers,Onno C. Meijer,Johan T. den Dunnen,Gert-Jan B. van Ommen,Annemieke Aartsma-Rus,Willeke M. C. van Roon-Mom +8 more
TL;DR: The results suggest that exon skipping may be a novel therapeutic approach to reduce polyglutamine-induced toxicity in spinocerebellar ataxia type 3.
Journal ArticleDOI
Ataxin-3 protein and RNA toxicity in spinocerebellar ataxia type 3: current insights and emerging therapeutic strategies.
TL;DR: The functioning of ataxin-3 and the involvement of known protein and RNA toxicity mechanisms of mutant ataxIn-3 that have been discovered are reviewed, as well as future opportunities for therapeutic intervention.