Results 311 to 320 of about 1,893,805 (368)

Single Administration of AAV‐mAtp6v1b2 Gene Therapy Rescues Hearing and Vestibular Disorders Caused by Atp6v1b2‐Induced Lysosomal Dysfunction in Hair Cells

open access: yesAdvanced Science, EarlyView.
Wei et al. establish a hair cell‐specific conditional knockout mouse model (Atp6v1b2fl/fl;Atoh1Cre/+), and demonstrate the importance of Atp6v1b2 for hair cell through maintaining the survival of lysosomes. A single administration of AAV‐ie‐Eh3‐mAtp6v1b2 through scala media at P0‐P2 realizes function compensation and restores hearing and balance ...
Gege Wei   +15 more
wiley   +1 more source

Transcriptomic Profiling Unveils EDN3+ Meningeal Fibroblasts as Key Players in Sturge‐Weber Syndrome Pathogenesis

open access: yesAdvanced Science, Volume 12, Issue 17, May 8, 2025.
Sturge‐Weber syndrome (SWS) is characterized by leptomeningeal vascular malformations, leading to seizures and stroke. Analysis of 119 446 brain cells from SWS patients uncovered distinct cell heterogeneity and identified an EDN3⁺ meningeal fibroblast cluster, with WNT5A emerging as a potential key driver of SWS progression and a promising therapeutic ...
Daosheng Ai   +14 more
wiley   +1 more source

α‐Synuclein Pathology Spreads in a Midbrain–Hindbrain Assembloid Model

open access: yesAdvanced Science, EarlyView.
A novel midbrain–hindbrain assembloid model demonstrates the spread of α‐synuclein pathology, a hallmark of Parkinson's disease, mimicking Braak's hypothesis. This model reveals how pathology propagates from the hindbrain to the midbrain, inducing synaptic changes and early signs of neuronal vulnerability. It offers an innovative platform for exploring
Gemma Gomez‐Giro   +14 more
wiley   +1 more source

The Role of Glia Telomere Dysfunction in the Pathogenesis of Central Nervous System Diseases. [PDF]

open access: yesMol Neurobiol
Papageorgakopoulou MA   +4 more
europepmc   +1 more source

Current progression in application of extracellular vesicles in central nervous system diseases. [PDF]

open access: yesEur J Med Res
Zhang XM   +9 more
europepmc   +1 more source

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