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This invention establishes a new approach to treating liver fibrosis using gene therapy.

Robust Genome Engineering in Primary Human T Cells using CRISPR/Cas9 Ribonucleoproteins

This invention enables highly effective experimental and therapeutic genomic engineering of primary human T cells and other hematopoietic cells with CRISPR/Cas9 ribonucleoprotein (RNP) technology.  

Novel Method of Using Modified and Optimized Bacterial-derived Genetic CRISPR System for Imaging, Regulating and Editing Mammalian Genomic Elements

This invention is a novel method using optimized small guide RNAs (sgRNAs) to enable dynamic imaging, editing and regulation of specific genomic elements in living mammalian cells via the CRISPR system.

Modular Cell and Drug Delivery Cannula System

The use of cell transplantation in the brain shows great promise for the treatment of human neurological diseases, such as Parkinson's disease or stroke. Indeed, pre-clinical studies in animal models have shown significantly improved neurological function following cell grafting. However, in human trials the results have been considerably more variable. This has, in part, been attributed to concerns with poor cell distribution within the target area. A further issue that has arisen with the challenge of scaling up from animal models to humans is the increase in the number of transcortical penetrations required to deliver therapeutic agents. For surgical cell transplantation approaches, cell sedimentation and impaired graft viability are also concerns that need to be addressed to optimize the use of this therapeutic avenue.

Enhanced gene activation through modification of small RNA duplexes

Small duplex RNAs have been shown to activate the expression of therapeutically relevant genes in a sequence-specific manner. UCSF researchers have identified chemical modifications and sequence features that enhance the activity and specificity of such duplex RNAs on targeted gene activation. 

Gene Therapy by Small Fragment Homologous Replacement

Gene therapy via viral vector technology has been associated with dangerous complications and risks. UCSF investigators have discovered a process that permits defective genetic sequences to be replaced with greater efficiency and potentially fewer side effects. The process, small fragment homologous replacement (SFHR), allows genes to be repaired in a site specific fashion and does not require the insertion of new genetic material into the genome. Thus, the SFHR approach should be applicable to a wide variety of gene therapy applications requiring the repair of specific mutations in DNA sequence. Furthermore, assay methods have been developed to monitor and quantify gene targeting frequency and to differentiate between cells carrying modified and unmodified DNA.

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