Posters

Validation of a luminometric assay for the detection of human anti-AAV neutralization factors.

A simple, rapid and robust bioluminescent assay for detecting anti-AAV neutralizing antibodies in serum samples

A highly sensitive nanoluciferase reporter enables efficient assessment of functional and genomic biodistribution of AAV in mice

Characterization of pre-existing anti-AAV T Cells using spectral flow cytometry in a cohort of healthy donors

Let's standardize our AAV biodistribution procedures for CNS gene therapy

Maximizing Upstream rAAV Yield: A Design of Experiments Approach to Plasmid Ratio Optimization

Generation and characterization of Xcell™ Eng-HEK293: a suspension cell line for rAAV production.

Surface-modified AAV6 vectors combined with nanoblades allow high level gene knock-in in hematopoietic stem and progenitor cells without compromising cell survival

Targeting a shared neoepitope derived from non-canonical translation of c-Myc oncogene in cancer cells

Development of 𝛾𝛿 T cells immunotherapy against glioblastoma

Systemic injection of recombinant AAV8 is able to rescue lysosomal acid lipase deficiency phenotype in a mouse model

Modelling therapeutic strategies in Duchenne muscular dystrophy by CRISPR/Cas-based approaches using iPSC-derived cardiomyocytes

Modeling fibrosis in duchenne muscular dystrophy organoids

ePsCas9 (eSpOT-ON): An Engineered High-Fidelity Genome Editor for Lipid Nanoparticle Delivery

Proof-of-concept for a one-step CRISPR-based gene therapy approach for STAT1 gain-of-function

TRPC3 inhibition reduces calcium entry in muscles of DMDmdx rats

Therapeutic research in dysferlinopathies: Progress in gene therapy

Identification of regulatory factors of FKRP gene expression in skeletal muscle

NewBiologix Xcell™ Genomic Analytical Platform: a novel platform for the genomic characterization of cell lines.

Discovery of New Therapeutic Targets for Duchenne Muscular Dystrophy by Inference of Gene Regulatory Networks

Improving AAV Purification : A Novel Affinity Chromatography Approach

Genetic reprogramming systems for spatiotemporally regulated expression of immune effectors by transferred B cells to cure chronic diseases

Optimising circular RNA production, expression efficiency, and delivery by viral pseudoparticles

Biodistribution and immune response against CNS-targeting AAV vectors in non-human primates

Development of a gene therapy based on SMaRT technology for Huntington's disease

Sponsor’s options for bioproduction site: advantages, risks and challenges