Small molecule splicing modifiers with systemic HTT-lowering activity
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Title
Small molecule splicing modifiers with systemic HTT-lowering activity
Authors
Keywords
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Journal
Nature Communications
Volume 12, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2021-12-15
DOI
10.1038/s41467-021-27157-z
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Note: Only part of the references are listed.- Huntingtin-Lowering Therapies for Huntington Disease
- (2020) Blair R. Leavitt et al. JAMA Neurology
- Risdiplam: First Approval
- (2020) Sohita Dhillon DRUGS
- Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy
- (2020) Shiori Ando et al. Scientific Reports
- Huntingtin Lowering Strategies for Disease Modification in Huntington’s Disease
- (2019) Sarah J. Tabrizi et al. NEURON
- Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
- (2019) Bryan Zeitler et al. NATURE MEDICINE
- Structural basis of a small molecule targeting RNA for a specific splicing correction
- (2019) Sébastien Campagne et al. Nature Chemical Biology
- Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
- (2018) Hasane Ratni et al. JOURNAL OF MEDICINAL CHEMISTRY
- Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease
- (2018) Amber L. Southwell et al. Science Translational Medicine
- Therapies targeting DNA and RNA in Huntington's disease
- (2017) Edward J Wild et al. LANCET NEUROLOGY
- Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
- (2017) Manaswini Sivaramakrishnan et al. Nature Communications
- Huntington's disease (HD): the neuropathology of a multisystem neurodegenerative disorder of the human brain
- (2016) U. Rüb et al. BRAIN PATHOLOGY
- Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy
- (2016) Hasane Ratni et al. JOURNAL OF MEDICINAL CHEMISTRY
- The Biology of Huntingtin
- (2016) Frédéric Saudou et al. NEURON
- Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis
- (2016) Guohao Wang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Treating the whole body in Huntington's disease
- (2015) Jeffrey B Carroll et al. LANCET NEUROLOGY
- SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice
- (2015) James Palacino et al. Nature Chemical Biology
- Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease
- (2014) Lisa M. Stanek et al. HUMAN GENE THERAPY
- SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
- (2014) N. A. Naryshkin et al. SCIENCE
- TopHat2: accurate alignment of transcriptomes in the presence of insertions, deletions and gene fusions
- (2013) Daehwan Kim et al. GENOME BIOLOGY
- STAR: ultrafast universal RNA-seq aligner
- (2012) Alexander Dobin et al. BIOINFORMATICS
- A fully humanized transgenic mouse model of Huntington disease
- (2012) Amber L. Southwell et al. HUMAN MOLECULAR GENETICS
- Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
- (2012) Holly B. Kordasiewicz et al. NEURON
- A Screen for Enhancers of Clearance Identifies Huntingtin as a Heat Shock Protein 90 (Hsp90) Client Protein
- (2011) Barbara Baldo et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Membrane transporters in drug development
- (2010) NATURE REVIEWS DRUG DISCOVERY
- Full-Length Human Mutant Huntingtin with a Stable Polyglutamine Repeat Can Elicit Progressive and Selective Neuropathogenesis in BACHD Mice
- (2008) M. Gray et al. JOURNAL OF NEUROSCIENCE
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