Abstract: Novel small molecule proteolysis-targeting chimeras (PROTACs) are provided, along with methods for their use as RIPK1 kinase degraders. The small molecule PROTACs described herein are useful in treating and/or preventing RIPK1 kinase-related diseases, such as cancer, neurodegenerative disorders, and inflammatory diseases. Also provided are methods for promoting RIPK1 kinase degradation in a cell using the compounds and compositions described herein.

Abstract: The present disclosure concerns hyperthermic devices for treating vascular involvements related to cancer therapies, such as surgery. In specific embodiments, the device is configured to provide therapeutic heating to destroy vessel-encasing tumors while still protecting the vessel itself. In particular embodiments, the devices utilize two opposing semi-cylindrical shells that encase the vessel in need of treatment of a tumor thereon. In other devices, a flexible substrate is guided under and around the vessel and tumor thereon.

Abstract: The present disclosure concerns systems and methods related to an adjustable surgical dilation device for the mechanical dilation of an anatomical opening, including, but not limited to, an endoscopic or robotic port. In specific embodiments, this device may create and expand an anatomical opening with a novel petal-like mechanism. In specific embodiments, this device may be utilized in laparoscopic procedures including, but not limited to, thyroid or parathyroid removal.

Abstract: The present disclosure relates to methods and compositions related to Natural Killer T cells that are engineered to harbor an expression construct that encodes an activator of the Wnt signaling pathway. Activation by expression of Wnt signaling activators or the addition of exogenous activators promotes NKT expansion over the course of multiple tumor cell challenges and improves long term tumor control in vitro. The present disclosure further includes NKT cells, populations, and methods to prepare them, that are engineered to express exogenous activators of Wnt signaling combined with chimeric antigen receptors (CARs) for therapeutic use.

Abstract: Embodiments of the disclosure include methods and compositions related to treating or reducing the severity or delaying the onset of one or more cardiac conditions in a mammal. In particular embodiments, the compositions concern Park2 and its use for a cardiac medical condition. In specific cases, effective amounts of Park2 polynucleotide(s) and/or Park2 polypeptide(s) are provided to an individual in need thereof, including for heart failure, for example. The administration may be locally to the heart, for example.

Abstract: MiniVectors and compositions containing MiniVectors that target genes implicated in IPF selected from CDH11, STAT3, STAT6, FoxM1, MDM2, TGF?, SMAD, PDGFA, or TLR4 and/or increase intracellular levels of reduced glutathione, relaxin, and p53, are provided, along with uses in the treatment of idiopathic pulmonary fibrosis.

Abstract: Methods and compositions used to identify and characterize novel rhodopsin domains, which are kalium (potassium)-conducting channelrhodopsins. The rhodopsin domain of these kalium (potassium)-conducting channelrhodopsins have been cloned, optimized and expressed in mammalian systems and thus may be used in, among others, optogenetic applications and as therapeutic agents for electrically active cell mediated disorders.

Abstract: MiniVectors and compositions containing MiniVectors that are ultrapure, and methods of making and using ultrapure MiniVectors for gene therapy uses, including long term repeated gene therapy uses.

Abstract: Embodiments of the disclosure concern methods of identifying whether or not antigens from a particular pathogen are immunogenic, including the order of their immunogenicity. Other embodiments concern correlations between attributes of T cells and their clinical efficacy, such as mathematical representations thereof.

Abstract: Embodiments of the disclosure concern systems, methods, and/or compositions for cultivation of mammalian viruses, including at least human noroviruses and sapoviruses within the Caliciviridae family of viruses. The ex vivo culture systems include intestinal enteroids in combination with bile or a functionally active fraction or component thereof. In specific embodiments, the culture system is utilized to test inactivation compounds for therapeutic or environmental efficacy and to test contaminated comestibles and/or environmental entities for determination of the presence of infectious virus. Furthermore, antiviral compositions may be tested using systems of the disclosure, including drugs, small molecule inhibitors, and biologics such as neutralizing monoclonal antibodies.

Abstract: Small molecules acting through the ligand binding domain of nuclear receptor TLX (NR2E1) are disclosed, as well as the use thereof for promoting neurogenesis and treating disorders associated with nuclear receptor TLX, including neurological disorders such as Alzheimer's disease. In certain embodiments, the invention provides compounds according to Formula II as described herein, and pharmaceutically acceptable salts or isomers thereof.

Abstract: The present disclosure concerns combination therapy for cancer that utilizes (i) an oncolytic virus; (ii) a virus comprising nucleic acid encoding an immunomodulatory factor; and (iii) at least one cell comprising a chimeric antigen receptor (CAR) specific for a cancer cell antigen. In particular embodiments, the virus comprises nucleic acid encoding an immunomodulatory factor comprises nucleic acid encoding IL-12 and/or antagonist anti-PD-L1 antibody.

Abstract: Disclosed is a handheld laser probe for laser thermal conjunctivoplasty, the handheld laser comprising: a forceps; and a line focused laser light source coupled to the forceps, wherein the forceps are configured to grasp a conjunctival fold and hold the fold in a light beam of the line focused laser, and wherein the line focused laser beam is configured to uniformly heat the conjunctival fold held in the forceps. Disclosed are systems for laser thermal conjunctivoplasty including the handheld laser. Disclosed are methods of conjunctivoplasty using the handheld laser probe.

Abstract: Provided are specific binding molecules, or fragments thereof, that bind to an epitope of IL13R?2, a receptor polypeptide preferentially found on the surface of cancer cells rather than healthy cells. Exemplary specific binding molecules are bispecific binding molecules that comprise a fragment of an IL13R?2 binding molecule and a peptide providing a second function providing a signaling function of the signaling domain of a T cell signaling protein, a peptide modulator of T cell activation, or an enzymatic component of a labeling system. Also provided are polynucleotides encoding such a specific binding molecule (e.g., bispecific binding molecule), vectors, host cells, pharmaceutical compositions and methods of preventing, treating or ameliorating a symptom associated with a cancer disease such as a solid tumor disease (e.g., glioblastoma multiforme).

Abstract: Systems and corresponding methods for an end-expandable optical fiber bundle are described herein. In one aspect, an endoscope can include a plurality of optical fibers each having a proximal end and a distal end; at least one camera coupled to the proximal ends of the plurality of optical fibers; and a sleeve enveloping the plurality of optical fibers proximate to the distal ends and repositionable along the length of the plurality of optical fibers, such that the sleeve is configured to control an angle of bend for each of the plurality of optical fibers as the optical fibers advance past the sleeve.

Abstract: Provided herein are compositions and methods for the synthesis, amplification, and in vitro transcription of full-length cDNA, or cDNA fragments. Methods are provided for reverse transcription of RNA and amplification for in vitro transcription. Further provided are method for loading of dendritic cells with the RNA and homologous lysate for immune stimulation.

Abstract: Compounds as STAT3 inhibitors are described. A pharmaceutical composition comprising the same, methods of making the same, and a method for treating or preventing conditions such as cancer, chronic inflammation, and fibrosis using the same, are described.

Abstract: Embodiments of the disclosure include certain formulations for methods of treating urea cycle disorders. The methods encompass compositions that comprise benzoate and phenylbutyrate that may be at certain doses and have certain ratios of the components. The benzoate and phenylbutyrate may act synergistically in treatment of the urea cycle disorders, in particular embodiments.

Abstract: An improved method of culturing cells for cell therapy applications that includes growing desired cells in the presence of antigen-presenting cells and/or feeder cells and with medium volume to surface area ratio of up to 1 ml/cm2 if the growth surface is not comprised of gas permeable material and up to 2 ml/cm2 if the growth surface is comprised of gas permeable material. The desired cells are at a surface density of less than 0.5×106 cells/cm2 at the onset of a production cycle, and the surface density of the desired cells plus the surface density of the antigen presenting cells and/or feeder cells are at least about 1.25×105 cells/cm2.

Abstract: The present invention features methods for characterizing mutational profiles in patients with bladder cancer.