Results 181 to 190 of about 24,572,306 (303)

Viral vectors : gene therapy and neuroscience applications /

open access: yes, 1995
Genetic manipulation of the adult mammalian nervous system is one of the most exciting areas in contemporary neurobiology. The explosive growth of this field has been facilitated by harnessing the power of viruses to transfer genetic material into ...
Loewy, Arthur D., Kaplitt, Michael G.
core  

Molecular Glue Degraders Enhance CAPRIN1‐Dependent Lysosomal Degradation of APP and Reduce Amyloid β in Alzheimer's Disease

open access: yesAdvanced Science, EarlyView.
A new class of lysosome‐directed molecular glue degraders selectively enhance CAPRIN1–APP interactions, driving APP degradation and reducing amyloid‐β production in human neurons and Alzheimer's disease mouse models. This CAPRIN1‐dependent targeted protein degradation strategy reveals a previously unrecognized therapeutic approach for disrupting the ...
Sunghan Jung   +15 more
wiley   +1 more source

Toward a Unified Neuroimmune Framework for Infection-Associated Psychiatric Disorders. [PDF]

open access: yesDiseases
Arbune M   +5 more
europepmc   +1 more source

Single‐Cell Profiling Reveals Clonally Expanded CX3CR1+ T Cells in Anti‐NMDA Receptor Encephalitis

open access: yesAdvanced Science, EarlyView.
CX3CR1+ T cells are associated with peripheral and central immune alterations in anti‐NMDA receptor encephalitis (NMDAR‐E). They show potential responsiveness to GluN1 (NR1) peptides, potential interactions with peripheral B cells, clonal expansion, increased CD137/CD154 expression, and inflammatory cytokine production. Their preferential cerebrospinal
Lin Yan   +13 more
wiley   +1 more source

Targeted Lysosomal Degradation of Extracellular and Membrane Proteins: From Receptor Hijacking to Programmable Endolysosomal Routing

open access: yesAdvanced Science, EarlyView.
Targeted chimeric degraders engage extracellular or membrane proteins and endocytosable cell‐surface receptors, forming ternary complexes that induce receptor‐mediated internalization. Following endocytosis, post‐endocytic sorting—not internalization alone—determines target fate.
Ke Liu   +6 more
wiley   +1 more source

CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome

open access: yesAdvanced Science, EarlyView.
By modeling Lowe syndrome using patient‐derived iPSCs, this study establishes a human disease model that faithfully recapitulates OCRL deficiency‐associated ciliary and cytoskeletal defects. The model enables evaluation of both mutation‐agnostic DNA augmentation and CRISPR‐mediated mutation correction strategies, both of which restore OCRL function and
Siyu Chen   +11 more
wiley   +1 more source

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