Amido-bridged nucleic acid (AmNA)-modified antisense oligonucleotides targeting α-synuclein as a novel therapy for Parkinson’s disease

Silencing Alpha Synuclein in Mature Nigral Neurons Results in Rapid Neuroinflammation and Subsequent Toxicity

(2018) Matthew J. Benskey et al.   Frontiers in Molecular Neuroscience

Synaptic regulator α-synuclein in dopaminergic fibers is essentially required for the maintenance of subependymal neural stem cells

(2017) Ana Perez-Villalba et al.   JOURNAL OF NEUROSCIENCE

Synucleins Have Multiple Effects on Presynaptic Architecture

(2017) Karina J. Vargas et al.   Cell Reports

Antisense Oligonucleotides Targeting Y-Box Binding Protein-1 Inhibit Tumor Angiogenesis by Downregulating Bcl-xL-VEGFR2/-Tie Axes

(2017) Kiyoko Setoguchi et al.   Molecular Therapy-Nucleic Acids

Intrathecal Injections in Children With Spinal Muscular Atrophy

(2016) Manon Haché et al.   JOURNAL OF CHILD NEUROLOGY

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

First-in-human assessment of PRX002, an anti-α-synuclein monoclonal antibody, in healthy volunteers

(2016) Dale B. Schenk et al.   MOVEMENT DISORDERS

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

shRNA targeting α-synuclein prevents neurodegeneration in a Parkinson’s disease model

(2015) Alevtina D. Zharikov et al.   JOURNAL OF CLINICAL INVESTIGATION

Amido-bridged nucleic acids with small hydrophobic residues enhance hepatic tropism of antisense oligonucleotides in vivo

(2015) Tsuyoshi Yamamoto et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Normalization of Overexpressed α-Synuclein Causing Parkinson's Disease By a Moderate Gene Silencing With RNA Interference

(2015) Masaki Takahashi et al.   Molecular Therapy-Nucleic Acids

Distribution of α-synuclein in the spinal cord and dorsal root ganglia in an autopsy cohort of elderly persons

(2015) Hiroyuki Sumikura et al.   Acta Neuropathologica Communications

The modern pre-levodopa era of Parkinson's disease: insights into motor complications from sub-Saharan Africa

(2014) R. Cilia et al.   BRAIN

Reducing C-Terminal-Truncated Alpha-Synuclein by Immunotherapy Attenuates Neurodegeneration and Propagation in Parkinson's Disease-Like Models

(2014) D. Games et al.   JOURNAL OF NEUROSCIENCE

Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides

(2014) Andrew D. Burdick et al.   NUCLEIC ACIDS RESEARCH

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

(2013) Michael E. Østergaard et al.   NUCLEIC ACIDS RESEARCH

Assessment of Sensorimotor Function in Mouse Models of Parkinson's Disease

(2013) Sheila M. Fleming et al.   Jove-Journal of Visualized Experiments

Amido-Bridged Nucleic Acids (AmNAs): Synthesis, Duplex Stability, Nuclease Resistance, and in Vitro Antisense Potency

(2012) Aiko Yahara et al.   CHEMBIOCHEM

A Progressive Mouse Model of Parkinson’s Disease: The Thy1-aSyn (“Line 61”) Mice

(2012) Marie-Francoise Chesselet et al.   Neurotherapeutics

Assessing the role of dopamine in limb and cranial-oromotor control in a rat model of Parkinson's disease

(2011) Jacqueline R. Kane et al.   JOURNAL OF COMMUNICATION DISORDERS

The Vermicelli and Capellini Handling Tests: Simple quantitative measures of dexterous forepaw function in rats and mice

(2010) Kelly A. Tennant et al.   Jove-Journal of Visualized Experiments

Antisense, RNAi, and gene silencing strategies for therapy: Mission possible or impossible?

(2008) E RAYBURN et al.   DRUG DISCOVERY TODAY

Incidence and Extent of Lewy Body-Related α-Synucleinopathy in Aging Human Olfactory Bulb

(2008) Renpei Sengoku et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

The Vermicelli Handling Test: A simple quantitative measure of dexterous forepaw function in rats

(2008) Rachel P. Allred et al.   JOURNAL OF NEUROSCIENCE METHODS

Patterns and stages of  -synucleinopathy: Relevance in a population-based cohort

(2008) J. Zaccai et al.   NEUROLOGY

Selective loss of nigral dopamine neurons induced by overexpression of truncated human α-synuclein in mice

(2006) Masaki Wakamatsu et al.   NEUROBIOLOGY OF AGING