Results 161 to 170 of about 73,809 (216)

Adeno-Associated Virus Toxicity in Duchenne Muscular Dystrophy: Mechanisms and Clinical Considerations. [PDF]

Genes (Basel)
Saylam E, D'ambrosio ES, Pesco MT, Gushchina LV.   +3 more
europepmc   +1 more source

Establishing Sensory Neurons as Therapeutic Targets in Peripheral Neuropathy Driven by Polyglutamine Expanded Murine ATXN3

Annals of Neurology, EarlyView.
Repeat expansion disorders frequently involve peripheral neuropathy, yet mechanisms remain unclear. Using a spinocerebellar ataxia type 3 (SCA3) Knock‐In Atxn3Q300/Q6, we identify progressive sensorimotor deficits, peripheral nerve pathology, and dorsal root ganglia RNA splicing dysregulation.
Juan P. Mato, John M. Hayes, Jacen Emerson, Maeha Sridharan, Toluwani Adelaja, Arsal Naeem, Alexandra F. Putka, Hayley S. McLoughlin   +7 more
wiley   +1 more source

Distinguishing Pseudotransduction and True Transduction Enables Characterization and Bioengineering of Extracellular Vesicle-Adeno-Associated Virus Vectors. [PDF]

J Extracell Vesicles
Boucher JD, Stranford DM, Edelstein HI, Tullman-Ercek D, Kamat NP, Leonard JN.   +5 more
europepmc   +1 more source

T Cell‐Mediated Targeting of Interneurons in Mice Shapes Hippocampal Remodeling and Epilepsy

Annals of Neurology, EarlyView.
Objective Autoimmune encephalitis (AE) is associated with autoantibodies targeting distinct neuronal populations. In AE, antibodies against glutamate decarboxylase 65 (GAD65), expressed in GABAergic interneurons, are frequently detected. In GAD65‐AE, hippocampal biopsies often show infiltrates of CD8+ cytotoxic T cells (CTLs), suggesting a prominent T ...
Daniel S. Galvis‐Montes, Lukas Henning, Karen M. J. van Loo, Rainer Surges, Susanne Schoch, Albert J. Becker, Julika Pitsch   +6 more
wiley   +1 more source

The adeno-associated virus Rep proteins target PP4:SMEK1 by preventing substrate recruitment. [PDF]

PLoS Pathog
Vandewinkel B, Torrekens S, Claes Z, Bollen M, Henckaerts E.   +4 more
europepmc   +1 more source

Improvement of Adeno-Associated Virus (AAV)-Based Technologies by Cell-Penetrating Penta-Peptides (CPP5s). [PDF]

Pharmaceutics
Guo CW, Diener A, Matsuyama S.
europepmc   +1 more source

Gene Therapy Using Recombinant Adeno-Associated Virus for Leber Congenital Amaurosis Induced by RPE65 Mutation. [PDF]

Clin Ophthalmol
Owliaee I, Teimoori A, Shoushtari M, Shojaeian A.   +3 more
europepmc   +1 more source

Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing

Animal Research and One Health, EarlyView.
Multiplex genome editing is transforming genome engineering from single‐gene perturbation to network‐level control, yet its broader application remains limited by challenges in gRNA array engineering, delivery technologies, and safety management. Emerging AI‐driven approaches are accelerating guide RNA design and CRISPR effector optimization for ...
Linli Wang, Yongbin Liu, Hongbing Han
wiley   +1 more source

Rewriting Renal Fate: The Evolving Landscape of Adeno-Associated Virus-Mediated Kidney Gene Therapies. [PDF]

Kidney360
Hickman PG, Asokan A, Wilson MH.
europepmc   +1 more source

Early synovial regulatory T cell engagement in posttraumatic osteoarthritis

Arthritis &Rheumatology, Accepted Article.
Objective Posttraumatic osteoarthritis (PTOA) develops despite restoration of joint biomechanics, suggesting that early post‐injury biological processes within the synovium shape disease trajectories. We investigated whether synovial regulatory T (Treg) cells are engaged during the early phase following joint injury and whether experimental enrichment ...
Xueyou Zhang, Mingde Cao, Jianting Li, Bruma Sai‐Chuen Fu, Michael Tim‐Yun Ong, Patrick Shu‐Hang Yung   +5 more
wiley   +1 more source