Results 261 to 270 of about 3,822 (305)

Unraveling 4‐Phenylbutyrate's Therapeutic Role in SLC6A1 Disorders: Pharmacochaperoning Over HDAC Inhibition

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Variants in SLC6A1, encoding the GABA transporter 1 (GAT‐1), cause epilepsy, autism spectrum disorder, and developmental delay via loss of GABA uptake, impaired trafficking, and ER retention. We previously found that 4‐Phenylbutyrate (PBA), an FDA‐approved drug, restores GABA uptake and reduces seizures in SLC6A1‐related disorders ...
Melissa B. DeLeeuw   +5 more
wiley   +1 more source

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito   +8 more
wiley   +1 more source

Floquet engineering of Feshbach resonances in ultracold gases. [PDF]

open access: yesSci Adv
Guthmann A   +4 more
europepmc   +1 more source

Uncovering G Protein‐Coupled Receptors: Novel Targets and Biomarkers for Predicting Glioma Prognosis

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Low‐grade gliomas (LGG) exhibit significant heterogeneity and recurrence risk. G protein‐coupled receptors (GPCR) contribute to glioma malignant progression, but their prognostic value remains unclear. This work attempts to formulate a GPCR‐based outcome‐predicting model for LGG. Methods Based on TCGA LGG data, the enrichment scores
Jun Yang   +4 more
wiley   +1 more source

Star Power: Early life stages of an endangered sea star are robust to current and near-future warming. [PDF]

open access: yesPLoS One
Hodin J   +9 more
europepmc   +1 more source

Multi-scale parameterization of neural rhythmicity with lagged Hilbert autocoherence. [PDF]

open access: yesImaging Neurosci (Camb)
Zhang S   +5 more
europepmc   +1 more source

Dose-Dependent Efficacy of a Riboflavin and Ultraviolet Light-Inactivated Whole-Virion SARS-CoV-2 Vaccine in a Hamster Infection Challenge Model. [PDF]

open access: yesVaccines (Basel)
Altina N   +14 more
europepmc   +1 more source

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