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
Characterization of Conformational Instability of Monoclonal Antibodies During Chromatographic Purification. [PDF]
Baran K, Podgórski R.
europepmc +1 more source
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
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]
Guthmann A +4 more
europepmc +1 more source
Uncovering G Protein‐Coupled Receptors: Novel Targets and Biomarkers for Predicting Glioma Prognosis
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
B<sub>0</sub> navigator enables respiratory motion navigation in radial stack-of-stars liver Look-Locker T<sub>1</sub> mapping. [PDF]
Stelter J +5 more
europepmc +1 more source
Star Power: Early life stages of an endangered sea star are robust to current and near-future warming. [PDF]
Hodin J +9 more
europepmc +1 more source
Observations of variable stars of long period
openaire +1 more source
Multi-scale parameterization of neural rhythmicity with lagged Hilbert autocoherence. [PDF]
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]
Altina N +14 more
europepmc +1 more source

