Abstract:
The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery particle formulations and/or systems comprising one or more components of a CRISPR-Cas system, which are means for targeting sites for delivery. The delivery particle formulations of the invention are preferably nanoparticle delivery formulations and/or systems. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
Abstract:
A glucose binding amphiphilic peptide hydrogel insulin delivery system that is responsive to glucose concentrations under physiological conditions is provided. Insulin is encapsulated in a glucose binding hydrogel, made from self-assembling amphiphilic peptides including a hydrophobic domain including a beta sheet forming region coupled to a charged hydrophilic domain modified to contain a glucose binding segment. The formulations are designed to release insulin as a function of blood glucose level, maintaining the patients' blood glucose level in an optimum range and avoiding both hyper- and hypoglycemia.
Abstract:
Biocompatible hydrogel capsules encapsulating mammalian cells having a diameter of greater than 1 mm, and optionally a cell free core, are disclosed which have reduced fibrotic overgrowth after implantation in a subject. Methods of treating a disease in a subject are also disclosed that involve administering a therapeutically effective amount of the disclosed encapsulated cells to the subject.
Abstract:
Water-responsive composite materials are provided containing a polymeric matrix and a water-responsive gel integrated into the polymeric matrix. The water-responsive gel can include a polyol or an alkoxylated polyol crosslinked by reversibly hydrolysable bonds, such as borate ester bonds. The polymeric matrix can include conjugated polymers such as poly(pyrrole) containing polymers. The composite material is capable of rapid actuation in the presence of a water gradient and can exhibit power densities greater than 1 W/kg. Methods of making water-responsive composite materials are provided, including by electropolymerization. Devices containing water-responsive composite materials are provided for sensing, locomotion, and power generation.