Author

J. N. Sleigh

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Open access Oct 2026

Combining a muscle-tropic AAV capsid with a muscle-specific promoter enhances skeletal muscle gene delivery while reducing off-target expression

Viral vector technologies based on adeno-associated viruses (AAVs) have demonstrated promising ability to deliver genetic cargo to a range of organs in vivo, with several novel candidates showing clinical efficacy in human trials over the past decade. However, naturally occurring AAV serotypes are limited in thei...

Annalucia Darbey, Wenanlan Jin, C. Spicer et al. · BMC Research Notes · 0 citations

Open access Sep 2026

Hyperactive KIF5A in Neurodegeneration.

The highly polarised morphology of neurons and the sheer length of their axons make transport of cargoes throughout the cell a formidable task. Decades of evidence obtained from genetic studies on patients and animal models highlight deficits in axonal transport as a recurrent cause, or early contributing factor, in a...

David Villarroel-Campos, J. N. Sleigh · Cytoskeleton · 0 citations

Open access Jul 2026

The neuropathy-causing GARS1ΔETAQ mutation drives pathology in subsets of motor and sensory neurons in mice

Together, these experiments show that GarsΔETAQ/+ mice display robust and selective peripheral nerve pathology that manifests in a general distal-to-proximal fashion, priming this CMT2D allele for testing treatments and evaluating mechanisms underlying peripheral nerve vulnerability.

R. Simkin, A. Paulo-Ramos, Qiuhan Lang et al. · bioRxiv · 0 citations