Publications by Project

Robust verification of genetic variant-associated candidate off-target sites

• Publication information coming soon

Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs

• Engineering stimuli-responsive extracellular vesicles for enhanced anticancer therapeutics
• Proteolysis Targeting Chimera Loaded Extracellular Vesicles for Developing Triple Negative Breast Cancer Treatment
  
• High-throughput Generation of Collagen Microbeads for Extracellular Vesicle Production and Therapeutic Delivery
• Extracellular vesicle-mediated gene editing for the treatment of nonsyndromic progressive hearing loss in adult mice

Validated Immunoassays to Accelerate Therapeutic Genome Editing INDs

• Tracer kinetic modeling of a 14 kDa anti-CD8 PET tracer (2C8v144) for in vivo estimation of CD8 expression in tissue and blood, binding affinity, and total body receptor occupancy

Utility of Human Organoids for Safety and Efficiency Evaluations of Genome Editing Therapeutics

• Calcium Shock Enables Efficient and Programmable Particle Delivery for Genome Editing Applications
• Imaging 3D cell cultures with optical microscopy
• Interferon-γ induces combined pyroptotic angiopathy and APOL1 expression in human kidney disease
• Recommendations on fit-for-purpose criteria to establish quality management for microphysiological systems and for monitoring their reproducibility
• Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease

A Modality-Agnostic Potency Assay Enabling Both Ex Vivo and In Vivo Genome Editing Therapeutics for Sickle Cell Disease

• Microfluidic capillary transit velocity as a functional measure for sickle cell disease and in vitro-derived red blood cells
• Measurement and clinical interpretation of CRISPR off-targets
• Motion blur microscopy: in vitro imaging of cell adhesion dynamics in whole blood flow
• A miniaturized wash-free microfluidic assay for electrical impedance-based assessment of red blood cell-mediated microvascular occlusion
• Rapid measurement of hemoglobin-oxygen dissociation by leveraging Bohr effect and Soret band bathochromic shift

Genome sequencing for evaluating the efficacy, specificity, and safety of human genome editing

• Regulatory Considerations for Genome-Edited T-cell Therapies

Optimization and qualification of biochemical CHANGE-seq unbiased genome-wide activity and gRNA sequencing methods for therapeutic genome editing INDs

• Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE
• Measurement and clinical interpretation of CRISPR off-targets
• Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice
• Virus-free CRISPR knockin of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells
• High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease
• Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease
• Selective haematological cancer eradication with preserved haematopoiesis
• Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

Optimization and qualification of cellular unbiased GUIDE-seq-2 genome-wide activity method for genome editing INDs

• Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE
• Measurement and clinical interpretation of CRISPR off-targets
• Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice
• Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning

Correction of Neurological Disease via Allele Specific Excision of Pathogenic Repeats

• A miniature CRISPR-Cas10 enzyme confers immunity by inhibitory signalling
• Measurement and clinical interpretation of CRISPR off-targets
• Characterizing and controlling CRISPR repair outcomes in nondividing human cells
• Directed evolution expands CRISPR-Cas12a genome-editing capacity
• Discovery research in physiologically maintained deceased
• nf-core/pacvar: a pipeline for analyzing long-read PacBio whole genome and repeat expansion sequencing data
• Single-molecule live-cell RNA imaging with CRISPR-Csm
• Functional protein mining with conformal guarantees
• CRISPR-Cas12a bends DNA to destabilize base pairs during target interrogation
• RNA language models predict mutations that improve RNA function
• Animal and bacterial viruses share conserved mechanisms of immune evasion
• Birth of protein folds and functions in the virome
• Zfp106 binds to G-quadruplex RNAs and inhibits RAN translation and formation of RNA foci caused by G4C2 repeats
• Rapid DNA unwinding accelerates genome editing by engineered CRISPR-Cas9
• Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excision
• Building CRISPR Gene Therapies for the Central Nervous System: A Review
• Engineering self-deliverable ribonucleoproteins for genome editing in the brain
• Validated assays for the quantification of C9orf72 human pathology
• Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection

Preclinical Genome Editing for Rare Neurological Diseases

• Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice
• Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference

Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases

• Implications of the FDA’s new plausible mechanism framework for the development of a personalized in vivo prime editing platform
• Measurement and clinical interpretation of CRISPR off-targets
• Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants
• How to create personalized gene editing platforms: Next steps toward interventional genetics
• Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs
• Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
• Preexisting maternal immunity to AAV but not Cas9 impairs in utero gene editing in mice
• Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need
• A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant
• Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing

The CRISPR Vision Program: Nonviral Genome Editing Platforms to Treat Inherited Retinal Channelopathies

• Integrating synthetic biology to understand and engineer the heart, lung, blood, and sleep systems
• Perspectives on the FDA platform technology designation program for the approval of gene therapies: a Swiss multi-stakeholder exploratory interview study
• Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader
• Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
• Identification of a Guide RNA Targeting an Ultraconserved Element for Evaluation of Cas9 Genome Editors Across Mammalian Species

Therapeutic Editing to Lower PrP in Prion Disease

• In vivo base editing extends lifespan of a humanized mouse model of prion disease
• Brainwide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editor
• An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery

Personalized Prime Editing as a Platform for Hepatic Inborn Errors of Metabolism

• Implications of the FDA’s new plausible mechanism framework for the development of a personalized in vivo prime editing platform
• Measurement and clinical interpretation of CRISPR off-targets
• Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants
• How to create personalized gene editing platforms: Next steps toward interventional genetics
• Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs
• Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

AAV-Mediated Editing to Treat Human Autosomal Dominant Hearing Loss DFNA41 and DFNA2

• Publication information coming soon

A Non-Viral CRISPR-Mediated Genome Editing Delivery Platform as as Potential Therapy for Neurogenic Diseases

• Publication information coming soon