Abstracts

Challenges for CRISPR-Cas9 genotoxicity: detection and prevention

POLYPRIME: A novel DNA-Polymerase based prime editing strategy

Zip Editing: an easy-to-use tool to increase CRISPR-Cas9 HDR-editing efficiency

Correcting Autoinflammation in STING-Associated Vasculopathy with Onset in Infancy (SAVI) by Human Stem Cell Genome-Editing

CRISPR activation of utrophin as a mutation-independent approach for Duchenne Muscular Dystrophy therapy

Efficient in vitro correction of a highly recurrent COL7A1 pathogenic variant using Cytosine Base editing to treat recessive dystrophic epidermolysis bullosa

CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy

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

AAV-mediated gene therapy of acid ceramidase deficiency by intravenous and CSF-directed vector administration

Gene therapy for familial hemophagocytic lymphohistiocytosis related to Munc 13-4 deficiency

In vivo CAR T cell therapy against angioimmunoblastic T cell lymphoma

Combination of TCR-deficient CAR-Tregs and non-mitogenic antiCD3 to promote transplant tolerance

A brain organoid model to study gene and cell therapy treatment for neuronopathic Gaucher disease

Modelling Stargardt disease using three-dimensional retinal organoids

Design of experiments assisted optimization of induced pluripotent stem cell (iPSC) directed differentiation toward airway progenitors for a cell and gene therapy against primary ciliary dyskinesia

3-year Follow-up of Crigler-Najjar Syndrome Patients Treated with AAV-based Gene Therapy in the GNT-012-CRIG Study 

Gene therapy for Artemis-SCID patients: preliminary results of the French ARTEGENE phase I/II clinical trial

Enhancing anti-tumoral properties of specific extracellular vesicles via miRNA loading: A novel approach for gene therapy in breast and pancreatic cancer models

Lentivector onco-targeting for solid tumor gene therapy

Universal allogeneic regulatory T cell therapy from genetically engineered iPSCs

Treg empowering through gene addition 

Skeletal muscle organoids for preclinical gene therapy with recombinant AAV vectors

Viral restriction factors impede the transduction efficiency of gene therapy lentiviral vectors and may be blocked to achieve therapeutic levels of gene transfer

Muscle-specific expression reduces early antigen presentation and promotes CD8 T cell tolerance after rAAV gene transfer

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