Modulation of host immunity in the airways with interferon lambda encoding mRNA

Affinity peptide mediated retargeting of AAV9 to cardiac interstitial cells.

In vivo endothelial cell gene silencing by siRNA-LNPs tuned with lipoamino bundle chemical and ligand targeting

Aerosolized gene and oligonucleotide therapy targeting microRNA-224 ameliorates pulmonary hypertension by orchestrating the BMP pathway 

Extracellular vaccine- or virus-derived SARS-CoV-2 spike protein: a potential link between reported pathologies.

Enhanced safety in multiplex-edited T cells through combined use of distinct CRISPR enzymes for knock-in and base editing

Peptide-assisted tethering of DNA repair effectors to Cas9 for precise genome editing

Translation of advanced cell therapies for HIV+ patients

CD3-zeta gene editing to reprogram T or NK cells with chimeric antigen receptors

An oncolytic HAdV-5 with reduced surface charge combines diminished toxicity and improved tumour targeting

Base editing restore cellular phenotype of T cells of patients with Hyper-IgE-Syndrome

Dual targeting of PD-L1 and ErbB2 by CAR-NK cells enables specific elimination of solid tumor cells and overcomes immune escape via antigen loss

ITR instability in E. coli? The answer is 42 (°C) for improving quantity and quality of rAAV

Cell Level Imaging of Nucleic Acid Therapeutics in Whole Mouse Bodies

Scalable generation of functional human iPSC-derived CAR-macrophages that efficiently eradicate CD19-positive leukemia

Third-generation lentiviral gene therapy rescues function in a mouse model of Usher 1B

Modifying immune responses to adeno-associate virus vectors by capsid engineering

Gene therapy approaches to potentially rescue catecholaminergic polymorphic ventricular tachycardia (CPVT)

Prime editing strategies for modeling and treatment of dilated cardiomyopathy 

Prime Editing for Correcting Familial Mutation Associated with Cardiomyopathy

Engineering miniature CRISPR-Cas proteins for gene editing in human cells

Establishing a targeted gene delivery platform for monogenic kidney disorders

Safety Support in (CAR)-T Cell Therapy Development:

- A qualitative qPCR assay as alternative approach for Replication Competent Lentivirus (RCL) monitoring -

Long-lasting antibody delivery by a myotropic Adeno-Associated Virus (AAV) Capsid Variant: high serum levels, favourable glycosylation profile and protection from SARS-CoV-2 challenge

Development of a New Therapeutic Approach by Genome Editing of Tenascin-C Expressed in Breast Tumor and Microenvironment

DART-AAVs enable specific transduction of human CD8 T cells for in vivo gene transfer

Evaluation of a novel mRNA trans-splicing dual AAV vector strategy to treat Usher syndrome 1B

Enhanced precision for in vivo gene delivery through bispecific AAV vectors towards targeting of HIV reservoir cells

Next-generation sequencing-guided screening of AAV9 peptide display libraries yields novel neurotropic capsids

Pleiotropic effects of AAV.SERCA2a treatment for heart failure in DMD pigs

Characterization of novel recombinant adeno-associated Virus (rAAV)-based capsid variants in the murine brain

T cells expressing HBV-specific chimeric antigen receptors harboring a Fab fragment control HBV infection in mice

Super-resolution microscopy reveals gene-transfer strategy-induced disparity of CAR expression affecting CAR-T cell function in an antigen density dependent manner

mRNA technology for the development of CAR T cells targeting fibrotic diseases

PSMA-specific natural killer cells for prostate cancer immunotherapy

Selective elimination of cancer cells in tissue culture by using the collateral activity of a novel CRISPR-Cas nuclease

Generation and analysis of cardiac progenitors expressing the DTPA-R PET-reporter gene for in vivo tracking of the cells in regenerative cell therapies

Identification and modeling of patient-specific CD19 escape variants to CAR T cell therapy

Enhancing endosomal escape of siRNA through chemical electron transfer

Base editing for monogenic kidney disease

CAR-T cells: a new strategy to fight cytomegalovirus infections in the immunocompromised host

Gene editing of MHC class I and II enhances allogeneic regulatory T cell therapy

Generation of enhanced CD19-specific CAR-T cells through identification and editing of exhaustion-associated genes

Investigating the epigenetic regulation of the β-globin switch to identify novel curative opportunities for β-hemoglobinopathies via targeted epigenome editing

CRISPR Prime Editing in Primary Human T Cell Subsets

Lipid-nanoparticle co-delivery of rAAV genomes and mRNA for AAV integrase Rep78 replicates wildtype AAV genome integration as assessed by targeted sequencing

CRISPR associated substrate-linked directed evolution (CaSLiDE) for evolving highly efficient and specific miniature CRISPR-Cas systems

Surveying sequence specificity of designer site-specific recombinases

A CRISPR-Cas9 double-hit strategy mitigates on-target aberrations and chromosomal translocations

Lipo-xenopeptide polyplexes for CRISPR Cas9 based gene editing at ultra-low dose

Base editing of hematopoietic stem cells restores immune function in a mouse model of familial hemophagocytic lymphohistiocytosis

Intein-mediated cis- and trans-splicing enables tighter control of Sleeping Beauty transposase activity

Enhancement of in vitro AAV-mediated gene transfer for bone regeneration using a cationic poloxamer

Universal CAST-Seq: Off-target detection of CRISPR nucleases and base editors

Targeting choroidal endothelial cells: Development of novel AAV variants for improved transduction

Next round in chemical evolution of xenopeptides for pDNA and mRNA transfer: The spacer effect

Novel AAV variants for improved transduction of retinal microglia

Next-generation transfection reagent for large scale therapeutic lentiviral vector production

pPLUS® AAV-Helper, novel engineered pHelper plasmid to improve yield and quality of several AAV serotypes in suspension cell culture systems. 

Transcriptome analysis supports specificity of multiplexed epigenome editing in primary T cells

Epigenome Editing Results in Successful Transient Reactivation of Gamma Globin Gene Expression and Offers an Innovative Therapeutic Option for Β-Hemoglobinopathies

siRNA CAPSULON Technology: Redefined siRNA Transfection

Evaluation of the Efficacy of Bcl-2-siRNA Loaded NK Cell-Based Exosomes in Xenograft Breast Cancer Model

Modeling delivery and efficiency of CRISPR systems in heart organoids

Amphiphilic balance: fine-tuning of double pH-responsive mRNA carriers towards higher potency

Optimizing miR-146a mimics: Unleashing precision in Graft-versus-Host Disease treatment

RNAi in macrophages mediated by efficient nanoparticles for siRNA delivery reveal complex effects on polarization markers upon knockdown of STAT3/STAT6

Fine-tuning immunity – ex vivo siRNA treatment to enhance efficiency of cell-based therapies

Control of bocavirus vector production by CRISPRa-VPR

rAAV displaying an anti-EGFR affibody transduces neuroendocrine tumor cell lines and organoids and mediates efficient enzyme prodrug tumor cell killing

RNA-switches facilitate drug-inducible expression of therapeutic proteins by oncolytic viruses

Measurement of endothelial force in vivo