Restoration, Not Bypass: Otoferlin Gene Therapy and a New Era in Hearing Loss Treatment
Abstract The FDA approval of Otarmeni, an AAV‐based gene therapy for biallelic OTOF‐associated sensorineural hearing loss, represents a conceptual shift in otology: from bypassing defective auditory physiology to restoring it. Unlike cochlear implantation, which circumvents damaged sensory structures, OTOF gene replacement targets a synaptic deficit in
Jazlyn A. Selvasingh, Justin R. Shinn
wiley +1 more source
Population dynamics of immunological synapse formation induced by bispecific T cell engagers predict clinical pharmacodynamics and treatment resistance. [PDF]
Liu C +5 more
europepmc +1 more source
Abstract Propionic acidemia (PA) is a neurometabolic disorder caused by propionyl‐CoA carboxylase deficiency with frequent neurological involvement, yet cell‐type‐specific mechanisms remain poorly defined. We established human induced pluripotent stem cell (iPSC)‐derived astrocytes (iAs) from patients harboring PCCA or PCCB mutations and investigated ...
Irene González‐Garnacho +5 more
wiley +1 more source
Clathrin mediates both internalization and vesicular release of triggered T cell receptor at the immunological synapse. [PDF]
Kvalvaag A +6 more
europepmc +1 more source
Microglia Ablation and Downstream Effects in the Cerebrospinal Fluid Proteome
ABSTRACT Disease‐related activation of glial cells leads to changes in the cerebrospinal fluid (CSF) proteome. However, assigning such protein changes to their cellular origins is often difficult. Here, we used microglia (and macrophage) depletion in mice to identify CSF proteins of microglial origin.
Sinja Buchner +8 more
wiley +1 more source
The Actin Cytoskeleton at the Immunological Synapse of Dendritic Cells. [PDF]
Rodríguez-Fernández JL +1 more
europepmc +1 more source
ABSTRACT Vascular dementia (VaD), accounting for up to 20% of dementia cases globally, is primarily driven by chronic cerebral hypoperfusion (CCH), which leads to progressive cognitive impairment. Despite the increasing prevalence of VaD, effective treatments remain limited.
Jung‐Hoon Kim, Yongtaek Oh, Suin Cho
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Transcriptional and post-transcriptional regulation of checkpoint genes on the tumour side of the immunological synapse. [PDF]
Dobosz P +3 more
europepmc +1 more source
ABSTRACT Diabetes‐related depression (DD) is a challenging comorbidity characterized by self‐reinforcing interactions between metabolic dysfunction and affective disturbance, while conventional antidepressants and glucose‐lowering drugs often yield suboptimal outcomes with adverse effects.
Shun Guo +5 more
wiley +1 more source
Learning from TCR Signaling and Immunological Synapse Assembly to Build New Chimeric Antigen Receptors (CARs). [PDF]
Cassioli C +3 more
europepmc +1 more source

