相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Engineered dual selection for directed evolution of SpCas9 PAM specificity
Gregory W. Goldberg et al.
NATURE COMMUNICATIONS (2021)
Double setback for ASO trials in Huntington disease
Katie Kingwell
NATURE REVIEWS DRUG DISCOVERY (2021)
CRISPR Interference-Potential Application in Retinal Disease
Caroline F. Peddle et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)
Huntingtin Lowering Strategies for Disease Modification in Huntington's Disease
Sarah J. Tabrizi et al.
NEURON (2019)
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease
Bryan Zeitler et al.
NATURE MEDICINE (2019)
CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model
Freja K. Ekman et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2019)
Theory of mind impairment in Huntington's disease patients and their relatives
Leo Bayliss et al.
ARQUIVOS DE NEURO-PSIQUIATRIA (2019)
Targeting Huntingtin in Patients with Huntington's Disease
Sarah I. Sheikh et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation
Ana M. Moreno et al.
MOLECULAR THERAPY (2018)
CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response
Emma Haapaniemi et al.
NATURE MEDICINE (2018)
CRISPR: Stressed about p53?
Miguel Foronda et al.
TRENDS IN MOLECULAR MEDICINE (2018)
p53 Throws CRISPR a Curve
Dana Carroll
TRENDS IN PHARMACOLOGICAL SCIENCES (2018)
CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease
Su Yang et al.
JOURNAL OF CLINICAL INVESTIGATION (2017)
Therapies targeting DNA and RNA in Huntington's disease (vol 16, pg 837, 2017)
E. J. Wild et al.
LANCET NEUROLOGY (2017)
Therapies targeting DNA and RNA in Huntington's disease
Edward J. Wild et al.
LANCET NEUROLOGY (2017)
Applications of CRISPR-Cas systems in neuroscience
Matthias Heidenreich et al.
NATURE REVIEWS NEUROSCIENCE (2016)
Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells
Daesik Kim et al.
NATURE BIOTECHNOLOGY (2016)
Differential Expression of Extracellular Matrix and Adhesion Molecules in Fetal-Origin Amniotic Epithelial Cells of Preeclamptic Pregnancy
Myung-Sun Kim et al.
PLOS ONE (2016)
Elucidation of Relevant Neuroinflammation Mechanisms Using Gene Expression Profiling in Patients with Amyotrophic Lateral Sclerosis
Yu Hui Won et al.
PLOS ONE (2016)
Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells
Daesik Kim et al.
NATURE METHODS (2015)
MicroRNA-124 slows down the progression of Huntington's disease by promoting neurogenesis in the striatum
Tian Liu et al.
NEURAL REGENERATION RESEARCH (2015)
Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases
Sangsu Bae et al.
BIOINFORMATICS (2014)
Development and Applications of CRISPR-Cas9 for Genome Engineering
Patrick D. Hsu et al.
CELL (2014)
Polyglutamine (PolyQ) Diseases: Genetics to Treatments
Hueng-Chuen Fan et al.
CELL TRANSPLANTATION (2014)
Preclinical and Clinical Investigations of Mood Stabilizers for Huntington's Disease: What Have We Learned?
Lisa Scheuing et al.
INTERNATIONAL JOURNAL OF BIOLOGICAL SCIENCES (2014)
Impaired TrkB Receptor Signaling Underlies Corticostriatal Dysfunction in Huntington's Disease
Joshua L. Plotkin et al.
NEURON (2014)
CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences
Yanni Lin et al.
NUCLEIC ACIDS RESEARCH (2014)
Comparison of non-canonical PAMs for CRISPR/Cas9-mediated DNA cleavage in human cells
Yilan Zhang et al.
SCIENTIFIC REPORTS (2014)
DNA targeting specificity of RNA-guided Cas9 nucleases
Patrick D. Hsu et al.
NATURE BIOTECHNOLOGY (2013)
Computational prediction of the PolyQ and CAG repeat spinocerebellar ataxia network based on sequence identity to untranslated regions
Jean L. Spence et al.
GENE (2012)
Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice
Mahmoud A. Pouladi et al.
HUMAN MOLECULAR GENETICS (2012)
Huntington's Disease and the Striatal Medium Spiny Neuron: Cell-Autonomous and Non-Cell-Autonomous Mechanisms of Disease
Michelle E. Ehrlich
NEUROTHERAPEUTICS (2012)
Conditional BDNF release under pathological conditions improves Huntington's disease pathology by delaying neuronal dysfunction
Albert Giralt et al.
MOLECULAR NEURODEGENERATION (2011)
Onset and Progression of Behavioral and Molecular Phenotypes in a Novel Congenic R6/2 Line Exhibiting Intergenerational CAG Repeat Stability
Randi-Michelle Cowin et al.
PLOS ONE (2011)
A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference
Maria Stella Lombardi et al.
EXPERIMENTAL NEUROLOGY (2009)
Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts
P. H. J. van Bilsen et al.
HUMAN GENE THERAPY (2008)
Wild-type huntingtin plays a role in brain development and neuronal survival
A Reiner et al.
MOLECULAR NEUROBIOLOGY (2003)
Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease
C Zuccato et al.
SCIENCE (2001)