Leszek Lisowski

Spatially precise neuron formation via hydrogel mediated modulation of the host astrocyte response

This paper develops a hydrogel-based system to reprogram astrocytes into neurons by delivering reprogramming factors and valproic acid, enhancing neural repair.

AAV2 Bypasses Direct Endosomal Escape by Using AAVR to Access the Trans-Golgi Network en Route to the Nucleus.

This study shows AAV2 bypasses endosomal escape by using AAVR to access the trans-Golgi network, challenging the model of direct endosomal escape.

Optimization of AAV tools to target Muller glial cells for retinal gene therapy

This study optimizes AAV serotypes and promoters to specifically target Muller glial cells in rats, identifying ShH10Y and GFAP promoter as the most effective combination for in vivo delivery.

Optimization of AAV tools to target Müller glial cells for retinal gene therapy

This study optimizes AAV serotypes and promoters to specifically target Müller glial cells in rats, identifying ShH10Y and GFAP promoter as the most effective combination for in vivo delivery.

Preferential vulnerability of cortical GABAergic interneurons to Nalcn deficiency

This study shows that NALCN deficiency preferentially affects GABAergic interneurons, reducing their intrinsic excitability and impairing contextual adaptation and spatial memory in mice.

Maturation of human cardiac organoids are required for disease modelling and drug discovery

This study shows that maturing human cardiac organoids enhances disease modeling and drug discovery by mimicking in vivo development and enabling therapeutic testing for DSP cardiomyopathy.

Characterisation of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina

This study characterizes the transcriptomic effects of Cas13bt3 in retinal cells and demonstrates its efficacy in silencing VEGFA mRNA in human retinal organoids and mouse models.

Alzheimer's disease induced neurons bearing PSEN1 mutations exhibit reduced excitability

This study investigates how PSEN1 mutations in Alzheimer's disease affect neuronal excitability using iPSC-derived neurons and finds reduced excitability and altered Kv/Nav channel currents in mutant neurons.

Harnessing the power of whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation

This study evaluates AAV vectors in human liver explants under normothermic perfusion to assess their functionality and neutralization by human sera.

A transient protein folding response targets aggregation in the early phase of TDP-43-mediated disease

This study identifies a transient protein folding response in early TDP-43 disease that reduces aggregation, with DNAJB5 as a protective factor, and shows proteomic similarities between mouse and human TDP-43 proteinopathies.

Proteome-wide systems genetics identifies UFMylation as a regulator of skeletal muscle function

The paper identifies UFMylation as a negative regulator of skeletal muscle function through proteome-wide systems genetics in mice and validates it in a disease model.