Gene Hung
ORCID：0000-0002-3840-1681
发表论文 87 篇 · 总被引 11668 次 · h-index 41
代表论文
- Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study (2016 · The Lancet · 被引 1044)
- Antisense oligonucleotide therapy for spinocerebellar ataxia type 2 (2017 · Nature · 被引 341)
- Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates (2014 · Journal of Pharmacology and Experimental Therapeutics · 被引 283)
- Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts (2015 · Nucleic Acids Research · 被引 213)
- Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron Disease Phenotypes in Spinal and Bulbar Muscular Atrophy (2014 · Neuron · 被引 180)
- Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy (2014 · Cell Reports · 被引 179)