Lack of sensorimotor toxicity when using the clinical SMN expression cassette in scAAV9在scAAV9中使用临床SMN表达盒未见感觉运动毒性Nature NeuroscienceIF202026-08-240PRE
A Role for SMN Exon 7 Splicing in the Selective Vulnerability of Motor Neurons in Spinal Muscular AtrophyMOLECULAR AND CELLULAR BIOLOGYIF2.72023-03-20107OAAI
RETRACTION: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN (Retraction of Vol 28, Pg 271, 2010)NATURE BIOTECHNOLOGYIF41.72022-10-064OA
Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic increment 7SMA miceNEUROBIOLOGY OF DISEASEIF5.62021-11-0119OAAI
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissueNATURE MEDICINEIF502021-10-0463PREAI
Intragenic complementation of amino and carboxy terminal SMN missense mutations can rescue Smn null miceHUMAN MOLECULAR GENETICSIF3.22020-10-214OAAI
Conditional deletion of SMN in cell culture identifies functional SMN allelesHUMAN MOLECULAR GENETICSIF3.22020-10-199OAAI
Complete sequencing of the SMN2 gene in SMA patients detects SMN gene deletion junctions and variants in SMN2 that modify the SMA phenotypeHUMAN GENETICSIF3.62019-02-2065OAAI
Mild SMN missense alleles are only functional in the presence of SMN2 in mammalsHUMAN MOLECULAR GENETICSIF3.22018-08-1013OAAI
The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophyNEUROBIOLOGY OF DISEASEIF5.62016-03-0146OAAI
Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMAHUMAN MOLECULAR GENETICSIF3.22015-08-1347OAAI
SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice (vol 24, pg 3847, 2015)HUMAN MOLECULAR GENETICSIF3.22015-07-299OAAI
SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMAHUMAN MOLECULAR GENETICSIF3.22015-07-2356OAAI
SMN deficiency disrupts gastrointestinal and enteric nervous system function in miceHUMAN MOLECULAR GENETICSIF3.22015-04-0945OAAI
Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose-response Study in Mice and Nonhuman PrimatesMOLECULAR THERAPYIF122015-03-01262OAAI
Electrophysiological biomarkers in spinal muscular atrophy: proof of conceptANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGYIF3.92013-12-0672OAAI
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouseHUMAN MOLECULAR GENETICSIF3.22011-12-20243OAAI
Temporal requirement for high SMN expression in SMA miceHUMAN MOLECULAR GENETICSIF3.22011-06-13124OAAI
Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN deliveryHUMAN MOLECULAR GENETICSIF3.22010-07-16200OAAI