Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME高级脊髓性肌萎缩症-肌阵挛性癫痫(SMA-PME)患者接受高剂量AAV9基因疗法后死亡Molecular TherapyIF122026-05-260OAAI
SUMOylation by SUMO2 is implicated in the degradation of misfolded ataxin-7 via RNF4 in SCA7 modelsDISEASE MODELS & MECHANISMSIF3.32019-01-1123OAAI
Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in MiceJOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGYIF32018-02-0216OAAI
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteinsMOLECULAR NEURODEGENERATIONIF17.52016-07-2811OAAI
Cross-talking noncoding RNAs contribute to cell-specific neurodegeneration in SCA7 (vol 21, pg 955, 2014)NATURE STRUCTURAL & MOLECULAR BIOLOGYIF10.12015-03-043OAAI
Cross-talking noncoding RNAs contribute to cell-specific neurodegeneration in SCA7NATURE STRUCTURAL & MOLECULAR BIOLOGYIF10.12014-10-1278OAAI
The autophagy/lysosome pathway is impaired in SCA7 patients and SCA7 knock-in miceACTA NEUROPATHOLOGICAIF9.32014-05-2452PRE
Interferon beta induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in miceBRAINIF11.72013-03-2160OA
Selective modulation of subtype III IP3R by Akt regulates ER Ca2+ release and apoptosisCELL DEATH & DISEASEIF9.62012-05-03147OAAI