Stanford researchers have developed a next-generation programmable transcriptional activation platform, TIGRa, that addresses key limitations of CRISPRa technologies, including large size, limited multiplexing capacity, and delivery constraints.
Stanford researchers have developed novel AsCas12a-expressing mouse models for simultaneous editing of multiple genomic loci in vivo with unique targeting capabilities relative to traditional Cas9 models, enabling the rapid creation of complex genotypes in somatic cells and ca
Stanford researchers have developed a novel CRISPR-based method, Oligo-LiveFISH, for generating large-scale pools of synthetic RNA oligos that enable multiplexed targeting, imaging, and manipulation of genomic regions in living cells.
Stanford researchers have developed a non-viral, homology-independent method for precise targeted DNA insertions into T-cells using electroporation and CRISPR/Cas9, enabling cost-effective production of CAR T-cells for T-cell therapies.
Stanford scientists have developed a gene integration system that uses human-derived helicases paired with CRISPR technology to enable precise insertion of long DNA sequences at targeted genomic locations.
Stanford researchers have developed a method to overcome the packaging capacity limitation of adeno-associated virus (AAV) Vector CRISPR/Cas9 systems to help treat genetic diseases for which the cargo is larger than 4.7kb.
Stanford researchers have developed AZD7648, a novel DNA-PK inhibitor that enhances HDR efficiency in CRISPR-Cas9 gene editing by shifting DNA repair from the error-prone NHEJ pathway to the precise HDR pathway, significantly improving gene targeting outcomes in human cells fo
Stanford researchers have discovered using a novel assay that a large proportion of CRISPR/AAV modified cells contain hidden concatemeric knockins that affect gene expression, and therefore developed a strategy to reduce their occurrence.
Stanford researchers have developed a new gene editing approach that enables red blood cell-specific gene expression for the treatment of enzyme deficiencies.
Genome editing of human hematopoietic stem and progenitor cells (HSPCs) has the potential to create a new class of medication for the treatment of inherited and acquired genetic diseases of the blood and immune system.
Stanford researchers have developed a new technology, Variant-FlowFISH, to enable high-throughput, highly sensitive measurements of how variants, introduced via CRISPR, affect gene expression.
Stanford researchers have developed novel technology that combines AAVMYO, a muscle cell targeting viral vector, with CRISPR base editors to achieve targeted gene repair, showcasing over 70% correction of hereditary mutations in cardiomyocytes.
To overcome current gene editing safety, efficacy, and scope limitations, Stanford researchers in the Mackall Lab and Stanley Qi Lab developed MEGA (Multiplexed Effector Guide Arrays), a versatile and multifunctional platform for programmable and scalable regulation of the T c
Researchers at Stanford have demonstrated the first method of its kind for treating cystic fibrosis (CF) using regenerated airway stem cells embedded on a biocompatible scaffold.
Researchers at Stanford have developed a potentially curative treatment strategy for alpha-thalassemia, one of the most common autosomal recessive disorders in the world involving the genes HBA1 and/or HBA2.