User profiles for Shibi Likhite
Shibi B LikhiteResearch Scientist, Nationwide Children's Hospital Verified email at nationwidechildrens.org Cited by 4740 |
[HTML][HTML] Single-dose gene-replacement therapy for spinal muscular atrophy
…, A Kaspar, S Corcoran, L Braun, S Likhite… - … England Journal of …, 2017 - Mass Medical Soc
Background Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor
neuron disease with an onset during infancy that results in failure to achieve motor milestones …
neuron disease with an onset during infancy that results in failure to achieve motor milestones …
Astrocytes from familial and sporadic ALS patients are toxic to motor neurons
Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease, with astrocytes implicated
as contributing substantially to motor neuron death in familial (F) ALS 1, 2, 3, 4, 5. However…
as contributing substantially to motor neuron death in familial (F) ALS 1, 2, 3, 4, 5. However…
Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS
Amyotrophic lateral sclerosis (ALS) causes motor neuron degeneration, paralysis, and death.
Accurate disease modeling, identifying disease mechanisms, and developing therapeutics …
Accurate disease modeling, identifying disease mechanisms, and developing therapeutics …
[HTML][HTML] Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose–response study in mice and nonhuman primates
…, L Schmelzer, L Braun, V McGovern, S Likhite… - Molecular Therapy, 2015 - cell.com
Spinal muscular atrophy (SMA) is the most frequent lethal genetic neurodegenerative disorder
in infants. The disease is caused by low abundance of the survival of motor neuron (SMN) …
in infants. The disease is caused by low abundance of the survival of motor neuron (SMN) …
[HTML][HTML] Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS
Mutations in superoxide dismutase 1 (SOD1) are linked to familial amyotrophic lateral
sclerosis (ALS) resulting in progressive motor neuron death through one or more acquired …
sclerosis (ALS) resulting in progressive motor neuron death through one or more acquired …
Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex
Sporadic amyotrophic lateral sclerosis (ALS) is a fatal disease with unknown etiology,
characterized by a progressive loss of motor neurons leading to paralysis and death typically …
characterized by a progressive loss of motor neurons leading to paralysis and death typically …
Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis
Astrocytes isolated from individuals with amyotrophic lateral sclerosis (ALS) are toxic to
motor neurons (MNs) and play a non–cell autonomous role in disease pathogenesis. The …
motor neurons (MNs) and play a non–cell autonomous role in disease pathogenesis. The …
[HTML][HTML] Neurotoxic astrocytes directly converted from sporadic and familial ALS patient fibroblasts reveal signature diversities and miR-146a theragnostic potential in …
A lack of stratification methods in patients with amyotrophic lateral sclerosis (ALS) is likely
implicated in therapeutic failures. Regional diversities and pathophysiological abnormalities in …
implicated in therapeutic failures. Regional diversities and pathophysiological abnormalities in …
[HTML][HTML] Gene therapy corrects brain and behavioral pathologies in CLN6-Batten disease
JT Cain, S Likhite, KA White, DJ Timm, SS Davis… - Molecular Therapy, 2019 - cell.com
CLN6-Batten disease, a form of neuronal ceroid lipofuscinosis is a rare lysosomal storage
disorder presenting with gradual declines in motor, visual, and cognitive abilities and early …
disorder presenting with gradual declines in motor, visual, and cognitive abilities and early …
[HTML][HTML] Translating SOD1 gene silencing toward the clinic: a highly efficacious, off-target-free, and biomarker-supported strategy for fALS
T Iannitti, JM Scarrott, S Likhite, IRP Coldicott… - … Therapy-Nucleic Acids, 2018 - cell.com
Of familial amyotrophic lateral sclerosis (fALS) cases, 20% are caused by mutations in the
gene encoding human cytosolic Cu/Zn superoxide dismutase (hSOD1). Efficient translation of …
gene encoding human cytosolic Cu/Zn superoxide dismutase (hSOD1). Efficient translation of …