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

• Publication information coming soon

Validated Immunoassays to Accelerate Therapeutic Genome Editing INDs

• Publication information coming soon

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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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