G. Hung

发表

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

R. Finkel, F. Rigo, D. Vivo, 2016, The Lancet.

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Y. Hua, A. Krainer, F. Rigo, 2011, Science Translational Medicine.

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

Daniel R. Scoles, S. Pulst, T. Otis, 2017, Nature.

Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.

S. Freier, M. Hayden, S. Warby, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Deletion or Inhibition of th e Oxygen Sensor PHD 1 Protects against Ischemic Stroke via Reprogramming of Neuronal Metabolism Graphical

P. Carmeliet, W. Robberecht, T. Dresselaers, 2016 .

Inhibition of Kirsten-Ras reduces fibrosis and protects against renal dysfunction in a mouse model of chronic folic acid nephropathy

B. Hendry, G. Hung, L. Newbury, 2019, Scientific Reports.

Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy.

Shuling Guo, B. Monia, Zhigang Yu, 2014, Cell reports.

Deficiency of the oxygen sensor prolyl hydroxylase 1 attenuates hypercholesterolaemia, atherosclerosis, and hyperglycaemia.

P. Carmeliet, L. Dubois, M. Daemen, 2016, European heart journal.

Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

Y. Hua, A. Krainer, N. York, 2014, The Journal of Pharmacology and Experimental Therapeutics.

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

H. Paulson, G. Hung, H. Kordasiewicz, 2017, Molecular therapy. Nucleic acids.

Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease

A. Messing, G. Hung, E. Swayze, 2018, Annals of neurology.

PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models

J. Svaren, S. Scherer, M. Scheideler, 2017, The Journal of clinical investigation.

Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant

S. Freier, M. Hayden, S. Warby, 2018 .

Inhibition of spinal cytosolic phospholipase A2 expression by an antisense oligonucleotide attenuates tissue injury-induced hyperalgesia

D. H. Kim, C. Svensson, B. Monia, 2008, Neuroscience.

Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy.

G. Sobue, C. Bennett, G. Hung, 2015, Human molecular genetics.

Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts

Christopher E. Hart, S. Freier, M. Sabripour, 2015, Nucleic acids research.

Viable RNaseH1 knockout mice show RNaseH1 is essential for R loop processing, mitochondrial and liver function

Christopher E. Hart, S. Crooke, S. Damle, 2016, Nucleic acids research.

Genomic Analysis of wig-1 Pathways

M. Sabripour, B. Monia, G. Hung, 2012, PloS one.

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

L. Shihabuddin, Seng H. Cheng, L. Stanek, 2012, Neuron.

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Y. Hua, A. Krainer, F. Rigo, 2010, Genes & development.

Correction of RNA Splicing with Antisense Oligonucleotides as a Therapeutic Strategy for a Neurodegenerative Disease

Y. Hua, A. Krainer, F. Rigo, 2012 .

Peripheral SMN restoration is essential for long-term rescue of a severe SMA mouse model

Adrian R. Krainer, Guy Horev, Y. Hua, 2011, Nature.

Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7

A. L. La Spada, C. Bennett, G. Hung, 2018, Science Translational Medicine.

Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

S. Freier, M. Hayden, S. Warby, 2014, PloS one.

Antisense oligonucleotide-mediated correction of transcriptional dysregulation is correlated with behavioral benefits in the YAC128 mouse model of Huntington's disease.

M. Hayden, L. Shihabuddin, Seng H. Cheng, 2013, Journal of Huntington's disease.

Rescue of Metabolic Alterations in AR113Q Skeletal Muscle by Peripheral Androgen Receptor Gene Silencing.

Y. Guan, Fan Zhu, M. Pennuto, 2016, Cell reports.

Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron Disease Phenotypes in Spinal and Bulbar Muscular Atrophy

A. Spada, C. Bennett, D. Cleveland, 2014, Neuron.

Pathological impact of SMN2 mis-splicing in adult SMA mice

Y. Hua, A. Krainer, F. Rigo, 2013, EMBO molecular medicine.

Enhanced Store-Operated Calcium Entry Leads to Striatal Synaptic Loss in a Huntington's Disease Mouse Model

I. Bezprozvanny, G. Hung, Jun Wu, 2016, The Journal of Neuroscience.

Deletion or Inhibition of the Oxygen Sensor PHD1 Protects against Ischemic Stroke via Reprogramming of Neuronal Metabolism.

P. Carmeliet, W. Robberecht, T. Dresselaers, 2016, Cell metabolism.

TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals.

Y. Hua, A. Krainer, G. Sobue, 2012, Genes & development.

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Y. Hua, A. Krainer, F. Rigo, 2010, Genes & development.

Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

Y. Hua, A. Krainer, N. York, 2014, The Journal of Pharmacology and Experimental Therapeutics.