Abstract: The invention relates to the use of precursor peptides and B1 proteins from lasso peptide biosynthesis systems for affinity purification, proximity-based sortase-mediated protein purification and ligation, and detection of fusion proteins. For proximity-based sortase-mediated protein purification and ligation, the invention relates to techniques that link protein purification with conjugation to other agents, including therapeutic agents, imaging agents, or linkers.

Abstract: DP-2 antagonists reversed PGD2-mediated human hair growth inhibition in a dose-dependent manner in vitro by reducing PGD2-triggered apoptosis and maintaining proliferation of keratinocytes. Hair follicles from approximately half of the alopecia patients exhibited little susceptibility to PGD2's effect in vitro. SNPs in the human DP-2 gene were identified that are associated with hair growth inhibition by PGD2. These findings underscore the role of DP-2 in regulating hair growth and indicate that DP-2 can be an effective approach in preventing and/or treating androgenetic alopecia in patients sensitive to PGD2. Furthermore, the SNPs identified here can be used to identify patients who will benefit from treatment.

Abstract: In one embodiment, a microfluidic device has a substrate that defines a first inlet for a continuous phase fluid, a second inlet for a dispersed phase fluid, and droplet generators that can produce micro-droplets from the continuous and dispersed phase fluids. The substrate defines (i) a plurality of delivery channels in fluid communication with the first and second inlets, each delivery channel having a first dimension along a first plane that is perpendicular to a transverse direction, and (ii) a plurality of trenches that extend from the delivery channels towards the droplet generators along the transverse direction. Each trench has a second dimension along a plane that is perpendicular to the transverse direction that is smaller than the first dimension. The substrate defines a plurality of vias that extend from the trenches to the droplet generators so as to fluidly connect the delivery channels with the droplet generators.

Abstract: This disclosure provides for compositions and methods that can be used to predict an individual's risk for experiencing a cardiovascular event following a surgical procedure (e.g., a non-cardiac surgical procedure).

Abstract: Methods for assessing efficacy of a treatment for a bestrophinopathy in a subject are provided. In certain embodiments, the subject has two mutant BEST1 alleles. In certain embodiments, the subject has at least one mutant BEST1 allele.

Abstract: Provided herein are devices for monitoring nucleic acid amplification, nucleic acid amplification monitors, methods of quantifying nucleic acid amplification, methods of identifying an unknown nucleic acid molecule, and systems for monitoring nucleic acid amplification. The disclosed devices, methods, and systems comprise at least one nuclemeter, comprising at least one sample chamber and at least one reaction-diffusion conduit in fluid communication with the chamber.

Abstract: The invention relates to proximity-based sortase-mediated protein purification and ligation. Specifically, the invention relates to techniques that links protein expression/purification with conjugation to therapeutic agents, imaging agents, or linkers.

Abstract: Provided are compositions and methods for treating diseases associated with expression of mesothelin comprising administering a cell that expresses a chimeric antigen receptor (CAR) specific to mesothelin in combination with a PD-L1 inhibitor.

Abstract: The presently disclosed subject matter provides native extracellular matrix-derived membrane inserts for organs-on-chips, multilayer microfluidics microdevices, bioreactors, tissue culture inserts, and two-dimensional and three-dimensional cell culture systems. A microfluidic cell culture is provided that can include at least one membrane including extracellular matrix (ECM) material. The ECM material can be used to construct a perfusable microfluidic system including a plurality of layers of microfabricated cell culture chambers. The microfluidic cell culture can further include a lower layer including a microchannel on which the at least one membrane is placed and an upper layer including another microchannel. The upper layer can be bonded to the lower layer.

Abstract: Disclosed herein are factor H variants, vectors comprising said factor H variants, and pharmaceutical compositions comprising the same for treatment of disorders.

Abstract: An optical switch device and a related method for defining a topological light transport channel in a photonic lattice are provided. An exemplary optical switch device includes a photonic lattice including a photonic topological microring array comprising a plurality of site rings coupled via a plurality of anti-resonant link rings, a plurality of input light ports and a plurality of output light ports located at the plurality of site rings, wherein the plurality of input light ports and the plurality of output light ports are respectively connected by a plurality of topological light transport channels. The optical switch device is further configured such that each of the topological light transport channels is defined by a gain domain area that is produced by a corresponding patterned optical pumping beam emitted onto the photonic topological microring array.

Abstract: Provided are compositions comprising phthalocyanine (PC)-loaded or naphthalocyanine (NC) dye-loaded nanoparticulate polymeric or lipid micelles and methods for visualization of tumors without optical imaging equipment, the methods comprising injecting a subject with the phthalocyanine (PC)-loaded nanoparticulate amphiphilic polymer or lipid micelles and b) detecting by endoscopy an accumulation of blue dye in a tumor and a demarcation of tumor margins. Also provided are methods for treating a tumor, the method comprising a) administering intravenously to a subject phthalocyanine (PC) dye-loaded or naphthalocyanine (NC) dye-loaded nanoparticulate amphiphilic polymer or lipid micelles; b) detecting by intraoperative photoacoustic imaging an accumulation of dye in a tumor and a demarcation of tumor margins; and/or c) irradiating the PC dye or the NC dye within the tumor with a laser to heat the tumor, thereby decreasing tumor cell viability and/or killing the tumor cells.

Abstract: Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional CDKL5 (hCDKLK5). Also provided are a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having CDD, or ameliorating symptoms of CDD, or delaying progression of CDD via administrating an effective amount of the rAAV to a subject in need thereof.

Abstract: Provided are Janus particles derived from natural starting materials, including starting materials that are plant-based and are not based on petrochemicals. Also provided are related compositions that include the disclosed particles, including emulsion compositions. Additionally provided are methods of synthesizing the disclosed Janus particles.

Abstract: Methods of treating GLA-deficiency (Fabry disease) in a subject in need thereof comprising administration of a recombinant adeno-associated virus (rAAV) vector having an AAV capsid and a vector genome comprising a sequence encoding a functional human alpha-galactosidase A (hGLA) are provided. Also provided are pharmaceutical compositions containing an rAAV vector and use of these pharmaceutical compositions for treating Fabry disease.

Abstract: The present disclosure relates, in part, to immune cell targeted lipid nanoparticle (LNP) compositions, and methods of use thereof for in vivo delivery of nucleic acid molecules and/or therapeutic agents to a target cell. In certain embodiments, the nucleic acid molecules encode chimeric antigen receptors (CARs). In certain embodiments, the target cell is a T cell. In certain embodiments, the present disclosure relates to the use of the LNPs described herein for the treatment, prevention, and/or amelioration of diseases and/or disorders, including but not limited to cancer.

Abstract: Compositions and methods are disclosed for treating cancer patients and identifying patients with a malignancy that are likely to respond to treatment with anti-PD-1/PD-L1 and other anti-cancer and immune-modulating therapeutics.

Abstract: A two-step chromatography purification scheme is described which selectively captures and isolates the genome-containing rAAV vector particles from the clarified, concentrated supernatant of a rAAV production cell culture. The process utilizes an affinity capture method performed at a high salt concentration followed by an anion exchange resin method performed at high pH to provide rAAV vector particles which are substantially free of rAAV intermediates.

Abstract: The present invention relates to compositions and methods for inducing an adaptive immune response against Zika virus (ZIKV) in a subject. In certain embodiments, the present invention provides a composition comprising a nucleoside-modified nucleic acid molecule encoding a ZIKV antigen, adjuvant, or a combination thereof. For example, in certain embodiments, the composition comprises a vaccine comprising a nucleoside-modified nucleic acid molecule encoding a ZIKV antigen, adjuvant, or a combination thereof.

Abstract: The invention provides T cells comprising nucleic acid sequence encoding a chimeric antigen receptor and a nucleic acid sequence encoding an enhancer of T cell priming, compositions including the T cells, and methods of using the T cells to treat diseases associated with the expression of disease-associated antigens.