Published in Mol Ther on January 06, 2014
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat Biotechnol (2016) 1.11
Oligonucleotide-based strategies to combat polyglutamine diseases. Nucleic Acids Res (2014) 0.90
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. Mol Ther (2015) 0.87
The role of the immune system in triplet repeat expansion diseases. Mediators Inflamm (2015) 0.85
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases. Hum Gene Ther (2016) 0.84
AAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancer. Proc Natl Acad Sci U S A (2015) 0.82
TRPC3 channels critically regulate hippocampal excitability and contextual fear memory. Behav Brain Res (2014) 0.81
Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia. Hum Mol Genet (2015) 0.80
Systemic delivery of adeno-associated viral vectors. Curr Opin Virol (2016) 0.78
Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease. Mol Ther Methods Clin Dev (2016) 0.77
Intravascular AAV9 Administration for Delivering RNA Silencing Constructs to the CNS and Periphery. Methods Mol Biol (2016) 0.76
Systemic injection of AAV9-GDNF provides modest functional improvements in the SOD1(G93A) ALS rat but has adverse side effects. Gene Ther (2017) 0.75
Gene suppression approaches to neurodegeneration. Alzheimers Res Ther (2017) 0.75
Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7. Nucleic Acids Res (2017) 0.75
Neuropathological classification of Huntington's disease. J Neuropathol Exp Neurol (1985) 7.93
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol (2008) 6.82
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci U S A (2005) 4.79
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A (2008) 4.53
Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron (2012) 4.48
Intranuclear inclusions and neuritic aggregates in transgenic mice expressing a mutant N-terminal fragment of huntingtin. Hum Mol Genet (1999) 4.06
Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data. Lancet Neurol (2009) 3.82
Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice. J Neurosci (2008) 3.54
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther (2010) 3.37
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther (2011) 2.91
Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood (2007) 2.70
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther (2009) 2.47
Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther (2005) 2.47
Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther (2012) 2.42
Mutant huntingtin causes context-dependent neurodegeneration in mice with Huntington's disease. J Neurosci (2003) 2.20
Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol (2009) 2.05
Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther (2003) 1.94
Beyond the brain: widespread pathology in Huntington's disease. Lancet Neurol (2009) 1.83
A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther (2009) 1.80
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Mol Ther (2011) 1.79
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol Ther (2011) 1.72
Widespread expression of the human and rat Huntington's disease gene in brain and nonneural tissues. Nat Genet (1993) 1.67
Weight loss in Huntington disease increases with higher CAG repeat number. Neurology (2008) 1.66
Creatine increase survival and delays motor symptoms in a transgenic animal model of Huntington's disease. Neurobiol Dis (2001) 1.57
Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Mol Ther (2012) 1.55
Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors. Mol Ther (2010) 1.38
Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice. Gene Ther (2009) 1.36
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Mol Ther (2013) 1.36
AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease. Mol Ther (2008) 1.31
Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome. J Neurosci (2013) 1.26
Assessment of the nutrition status of patients with Huntington's disease. Nutrition (2004) 1.20
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. Mol Ther (2012) 1.16
Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Mol Ther (2013) 1.16
Mutant huntingtin causes metabolic imbalance by disruption of hypothalamic neurocircuits. Cell Metab (2011) 1.14
Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol Ther (2011) 1.09
Therapeutic cardiac-targeted delivery of miR-1 reverses pressure overload-induced cardiac hypertrophy and attenuates pathological remodeling. J Am Heart Assoc (2013) 1.08
Neuroendocrine disturbances in Huntington's disease. PLoS One (2009) 1.06
Structural MRI detects progressive regional brain atrophy and neuroprotective effects in N171-82Q Huntington's disease mouse model. Neuroimage (2011) 1.05
Neuronal loss in the hippocampus in Huntington's disease: a comparison with HIV infection. J Neurol Neurosurg Psychiatry (1993) 1.05
Cortical and striatal neurone number in Huntington's disease. Acta Neuropathol (1994) 1.00
Viral delivery of glial cell line-derived neurotrophic factor improves behavior and protects striatal neurons in a mouse model of Huntington's disease. Proc Natl Acad Sci U S A (2006) 0.96
Changes in key hypothalamic neuropeptide populations in Huntington disease revealed by neuropathological analyses. Acta Neuropathol (2010) 0.96
Gene therapy for Huntington's disease. Neurobiol Dis (2011) 0.94
Neuronal distribution of intranuclear inclusions in Huntington's disease with adult onset. Neuroreport (1998) 0.94
Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery. Gene Ther (2013) 0.93
Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of α-dystroglycan and improves muscle functions. Mol Ther (2013) 0.93
Cerebellar atrophy in Huntington's disease. J Neurol Sci (1981) 0.84
NGF and nitrosative stress in patients with Huntington's disease. J Neurol Sci (2012) 0.81
The spectrum of motor disorders in Huntington's disease. Clin Neurol Neurosurg (1992) 0.76