Stanford researchers have developed a high-sensitivity cell-based assay for predicting the innate immune response to recombinant adeno-associated virus.
Researchers in Prof. Mark Kay's laboratory have developed variant AAV (adeno-associated virus) vectors with specificity and high transduction efficiency for pancreatic alpha- and beta- islet cells.
Stanford researchers have invented a method and developed compositions of matter to reduce the production of infectious viruses in cells that line the respiratory tract. The invention enables the use of gene-silencing approaches to prevent and treat viral infections.
The Hu Lab at Stanford has developed a neuroprotective gene therapy for treating glaucoma and other optic neuropathies. Their gene therapy AAV vector expresses NMNAT2 operably linked to a retinal ganglion cell-specific promoter (mSngc).
Researchers at Stanford have developed a CRISPR-based system to degrade viral RNA, with potential applications as both an anti-viral therapeutic and a prophylactic treatment against influenza, SARS-CoV-2, and other viruses.
Adeno-associated virus (AAV) vectored products are currently leading candidates for gene therapy applications with multiple approved products and many more in clinical trials.
Researchers at Stanford University have developed a method to induce antigen-specific immunological tolerance to allow for more effective gene replacement therapy for Duchenne muscular dystrophy (DMD).
Researchers in the Mark Kay group have identified a method to increase the rates of AAV vector-mediated HR (AAV-HR) in mammalian cells, increasing the efficiency of gene targeting rate
Stanford researchers have developed a gene therapy that combines a retinal ganglion cell (RGC)- specific promoter with CRISPR gene editing to provide effective neuroprotection in optic neuropathies.
Researchers at Stanford have developed a new therapeutic to promote survival of retinal ganglion cells (RGCs) and optic nerve regeneration after traumatic injury or optic neuropathies.
Researchers at Stanford have developed methods to overcome the limited packaging capacity of adeno-associated virus (AAV) vectors and enable their use in integration of large transgenes.
Researchers in Prof. Mark Kay's laboratory have continued to develop novel recombinant adeno-associated viral (AAV) capsids via capsid gene shuffling that transduce human hepatocytes at high efficiency in vivo.
Researchers in Prof. Mark Kay's laboratory have developed recombinant adeno-associated viral (AAV) capsid proteins that transduce human primary hepatocytes at high efficiency in vitro and in vivo.
Researchers from Dr. Mark Kay's laboratory at Stanford University have merged desirable qualities of multiple natural AAV isolates by an adapted DNA family shuffling technology to create a complex library of hybrid capsids from eight different wild-type viruses.