Researchers at Stanford have developed a new catalog of compact transcription effector domains and fused them onto DNA binding domains to engineer synthetic transcription factors.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an efficacious therapy for patients with life-threatening leukemias, but its use has been hindered by the limited availability of donors with matching HLA. Graft manipulation by removing ??
RNA replication and amplification have broad applications across biomedicine, but current methods are limited by a reliance on inefficient, multi-step protocols.
Researchers in Prof. Stephen Quake's laboratory have developed a CRISPER-Cas-based targeted endonuclease system designed to treat latent viral infections by attacking the viral genome.
Researchers at Stanford have developed a novel therapeutic approach for dilated cardiomyopathy (DCM). DCM is characterized by left ventricular enlargement and reduced systolic function.
Stanford researchers have developed a new class of materials that enable new strategies for the efficient delivery of messenger RNA (mRNA) into cells and animals. The delivery materials are easily prepared (2 steps), stable and readily tuned.
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.
Dr. Mark Kay and colleagues have created antibiotic-selectable, non-silencing plasmid vectors that can be prepared by conventional methods and provide persistent high levels of transgene expression.
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.
Stanford researchers have patented protein stability regulation methods using destabilizing domains (DDs) optimized for use in humans. The ability to control specific protein abundance in cells is a powerful tool for gene therapy and investigating biological behavior.
Stanford researchers have developed a method that can tune the ratio between reversible (RE) and irreversible (IRE) electroporation through waveform adjustments.
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.