Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

Yimin Hua, Kentaro Sahashi, Frank Rigo, +4

Nature

Regenerative MedicineNeuroregenerationSpinal Cord InjuryPeripheral Nerve InjuryLong-term Rescue +7

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

Yimin Hua, Kentaro Sahashi, Gene Hung, +4

Genes & Development

ImmunologyCell DeathPathologyMolecular BiologyCytoskeleton +16

Strong correlation between the number of CAG repeats in androgen receptor genes and the clinical onset of features of spinal and bulbar muscular atrophy

S. Igarashi, Yoshinori Tanno, Osamu Onodera, +9

Neurology

Androgen Receptor GenesRare DiseasesDevelopmental BiologyCag RepeatsMotor Neuron Disease +13

Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models

Yimin Hua, Ying Hsiu Liu, Kentaro Sahashi, +3

Genes & Development

Knockout MouseNeurodegenerative DiseasesAmyotrophic Lateral SclerosisSpinal Cord InjuryComplementary Decoy Oligonucleotide +12

In vitro and in silico analysis reveals an efficient algorithm to predict the splicing consequences of mutations at the 5′ splice sites

Kentaro Sahashi, Akio Masuda, Tohru Matsuura, +6

Nucleic Acids Research

Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice

Rahul Sinha, Young Jin Kim, Tomoki Nomakuchi, +5

Nucleic Acids Research

Pathological impact of <scp> <i>SMN</i> </scp> <i>2</i> mis‐splicing in adult <scp>SMA</scp> mice

Kentaro Sahashi, Karen Ling, Yimin Hua, +10

EMBO Molecular Medicine

Knockout MouseRare DiseasesPathological ImpactGenetic DisorderGenetics +13

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

Kentaro Sahashi, Masahisa Katsuno, Gene Hung, +7

Human Molecular Genetics

Androgen ReceptorSocial SciencesSkeletal MuscleNeuroendocrine MechanismDegenerative Pathology +16

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

Kentaro Sahashi, Yimin Hua, Karen Ling, +8

Genes & Development

NeurogenomicsMotor Neuron DiseasePathologyTherapeuticsSynaptic Signaling +18

NUS1 mutation in a family with epilepsy, cerebellar ataxia, and tremor

Kunihiko Araki, Ryoichi Nakamura, Daisuke Ito, +10

Epilepsy Research

Mendelian DisorderNeurological DisorderGenetic DisorderGeneticsDegenerative Disease +9