Comprehensive Interrogation of Synthetic Lethality in the Human DNA Damage Response

Programmable recombinases to treat genetic diseases

The ethics of human genome editing

Establishment of a stable episomal replicon system for HBV to screen novel RNA-based antiviral gene therapies

A direct comparison of the Sleeping Beauty Transposon system and the CRISPR/Cas9 machinery for genetic engineering

Using High-Throughput Genetic Screening to Improve Stable AAV Producer Cell Lines and to enhance viral vector production

Programmable genome disruption enables selective elimination of cancer cells using a novel CRISPR-Cas nuclease

‘One-pot’ PASTA - Advanced T cell Engineering through Precise Integration of Super-Large Exogenous DNA

Gene editing for pre-leukemia bone failure syndrome, severe congenital neutropenia, – current advances and critical considerations

CRISPR-edited allogeneic BCMA-CAR NK cells to overcome loss-of-target in multiple myeloma

AAV-mediated gene therapy in a porcine model of Duchenne Muscular Dystrophy

An enhanced Single-Strand Break-Based Strategy for safer and more efficient Genome Editing

CAR-NK cell therapies for treatment of severe autoimmune diseases

Distinct preferences of CD8⁺ and CD4⁺ CAR-T cells for transcription factor enhancement

Zebrafish as a novel in vivo model to assess CAR-NK cell efficacy against metastatic breast cancer

Development of a Broad-Spectrum siRNA Therapy Against Human Coronaviruses

IRON: IRES launched RNA replicon for versatile and efficient gene expression

RNAvolution in CAR-T cell therapy - Optimising RNA to generate transient CAR-T cells

An all-in-one approach for mutations in STAT3 causing Hyper-IgE-syndrome

Generation of mRNA-based therapeutic vaccines against chronic HBV infection

A base editor mediated knock-in (BEKI) system for the non-viral generation of multiplex gene edited CAR T cells

UCAST: Universal detection of off-target effects of base editors

An ex vivo CRISPR/Cas9 based homology-directed repair gene therapy for p.W44X mutations in the HAX1 gene

AAV-Mediated Prime Editing for the correction of Familial Mutations Associated with Cardiomyopathy

Enhancing CRISPR/Cas9 Gene Therapy: Chemical Modulators for Improved Safety and Precision

Modulating C-to-T editing with TALE base editors

EDSpliCE: AAV-deliverable enhanced deletion RNA-guided nucleases for therapeutic splicing modulation in USH2A  

Design and test of a prime editor and LNPs for the CFTR F508 deletion in lung cells

VꮋH-based CLEC12A-CAR-NK cells based on llama nanobodies for treatment of Acute Myeloid Leukemia

Peptide-assisted tethering of critical DNA repair effectors to Cas9 enables precise genome editing in Fanconi anemia

CRISPR Therapeutic Strategy for Stargardt Disease: Correcting Exonic ABCA4:c.768G>T Mis-splicing.

Efficient Single-Base Substitution with HDR-Cas9: A Step Towards Sickle Cell Disease Cure

Developing Designer-Recombinase induced Gene Replacement (DRiGR) with clinical implications

A base editing platform for the correction of cancer driver mutations unmasks conserved p53 transcription programs

Towards a prime-boost vaccine platform based on different adenovirus types  

AAV's next top model: Influence of animal species on directed evolution of myotropic AAV capsids

CAPSULON: The Next Step in Efficient and Safe siRNA Delivery for Clinical Applications and Gene Therapy

Bypassing ex vivo CAR T cell manufacturing: leveraging LNPs for in vivo delivery

Versatile lipid nanoparticle platform for efficient CRISPR-Cas9 gene editing in primary T cells and CD34+ hematopoietic stem cells

Genome-Scale CRISPR/Cas9 screening in tumor cells to identify mechanisms for overcoming cancer resistance in NK cell-based immunotherapy

Shortened Half-Life Sleeping Beauty Transposase for Biosafety-Enhanced CAR-T Cell Production

FindMe2care – a contact platform for patients with genetically confirmed diagnoses