Posters

Mtm1 deficient rats as a new preclinical model for myotubular myopathy gene therapy

A la recherche des enhancers inconnus: identifying satellite cell-specific enhancers for targeted gene therapy

Preclinical evaluation of AAV-U7snRNA Exon Skipping Targeting exon 44 and exons 6--8 demonstrates dystrophin rescue and functional benefit in humanized DMD mouse models

AAV cellular response is mediated by CD8⁺ effector memory T cells in healthy donors

CRISPR/Cas9 and AAV-mediated gene correction approach for B-hemoglobinopathies

Stargardt Therapeutic ABCA4 Restoration: proof-of- concept for the ABCA4 gene and Stargardt disease

Bioconjugation of the capsid of adeno-associated viruses for osteoarthritis treatment by gene therapy

Assessment of the Innate anti-AAVr immune response in human blood

Tropism and underlying mechanisms of 2nd generation AAV vectors for neuromuscular diseases

AAV Viability following highly diverse 7mer insertions

Alternative delivery of adeno-associated virus 9 for the treatment of Duchenne muscular dystrophy to target CSF and muscles-- GFP Biodistribution study in WT mice

Immune tolerance to hepatic antigens involve a dysfunctional Th1-like CD4+ T cell subset

A novel suprachoroidal AAV vector engineered with ALIGATERᵀᴹ achieves unprecedent posterior segment targeting in non-human primates

Gene Therapy For Spinocerebellar Ataxia 7 : Restoring Cholesterol Metabolism

Novel intravenous AAV gene therapy for mucopolysaccharidosis type IIIA and IIIB in mouse and canine model of the pathology

Therapeutic challenges in Glycogen Storage Disease type III: how proliferation and inflammation influence rAAV gene transfer stability in a mild fibrotic background

TRPC3 inhibition reduces calcium entry in muscles of DMDmdx rats

Development of immunocompetent skeletal muscle organoids to study T-cell mediated anti-AAV cellular immune response

Evaluation of a universal reference gene for AAV biodistribution in small and large animal models

Spliceosome-mediated RNA trans-splicing (SMaRT) gene therapy for Stargardt disease

Novel ligand-conjugated AAV vectors enable precise TfR1-mediated retargeting to the CNS after systemic delivery

Viral safety by design: Addressing unique challenges in gene therapy development

Systemic AAV vector readministration by combination of natural and bioengineered capsids

Exon skipping for the second Calponin Homology Domain of dystrophin using AAV.U7snRNA - In vitro & Intramuscular studies using a novel murine model of Duchenne Muscular Dystrophy

Intensification of rAAV Purification at the Capture Step Using Multi-Column Chromatography with the Resolute® BioSMB PD System

Establishing a robust and open-access rAAV manufacturing process for gene therapy within a major European partnership (ERDERA)

Conditioning strategies to enhance functional maturation of human EMT (Engineered Muscle Tissues)

Streamlined Production of 105 Barcoded rAAV Capsid Variants for Pooled Evaluation in Non-Human Primate Retina

Development of a High-Yield AAV Production Platform Using DOE and Automated Bioreactors

Advanced pipeline for CRISPR/Cas9 off-targets detection in Guide-seq and related integration-based assays