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Invited Speaker
Muscle repair potential of adult stem cells through different modes of action
Other
Skeletal muscle organoids for preclinical gene therapy with recombinant AAV vectors
Modelling Stargardt disease using three-dimensional retinal organoids
Cell Therapy
Combined cellular and gene therapy to treat primary ciliary dyskinesia
High systemic dosing of AAV 9 vectors: a relevant rat model to study the impact of vector immunogenicity in the liver
Gene Editing
NHEJ inhibition increases homology-mediated AAV integration in hematopoietic cells
Study of retinal microglial cells after sub-retinal injection of recombinant AAV in pigs
Development of an AAV bioproduction technological platform in microalgae
Treatment of myotonic dystrophy type 1 with CRISPR/Cas9
Mutation independent CRISPR/Cas9-induced allele deletion results in vitro in a functional benefit for dominant RYR1 mutation
Homology-independent targeted integration (HITI) as possible treatment for Stargardt disease.
Delivery of Cas9 ribonucleoprotein to the retina of mice with Retinitis pigmentosa
New technologies for CAR-T characterization and potency testing
Multiomic analyses reveals epigenome remodelling and transcriptional co-factors as critical determinants for neuronal cell therapy
Can academic structures improve access to CAR-T cells?
A novel technology for accurate quantification of viral genome and determination of lipid nanoparticle encapsulation efficiency
Human myoblasts and FAPs cell lines: easy-to-use and genetically modular models to study neuromuscular diseases
Genome editing for RyR-1 related myopathies
Dystrophin deficiency impairs cell junction formation during embryonic myogenesis
AAV9 viral vector genome engineering with TLR9 DNA-based immunomodulating sequences improves gene transfer efficiency after high systemic dosing
