Abstract: Nucleic acid molecules and compositions comprising one or more nucleic acid sequences that encode a consensus filovirus immunogen including a consensus Marburgvirus filovirus glycoprotein MARV GP immunogen, a consensus Ebolavirus Sudan filovirus glycoprotein SEBOV GP immunogen and a consensus Ebolavirus Zaire glycoprotein ZEBOV GP immunogen are disclosed. The coding sequences optionally include operable linked coding sequence that encode a signal peptide. Immunomodulatory methods and methods of inducing an immune response against filovirus, particularly Marburgvirus, Ebolavirus Sudan and Ebolavirus Zaire are disclosed. Method of preventing filovirus infection, particularly infection by Marburgvirus, Ebolavirus Sudan and Ebolavirus Zaire and methods of treating individuals infected with filovirus infection, particularly infection by Marburgvirus, Ebolavirus Sudan and Ebolavirus Zaire are disclosed. Consensus filovirus proteins are disclosed.

Abstract: Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5? inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional human N-acetyl-alpha-glucosaminidase (hNAGLU), a regulatory sequence which direct expression of hNAGLU in a target cell, and an AAV 3? ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIB.

Abstract: A cell bag rotator is constructed to slowly agitate a plurality of cell bags by rotation along a rotation axis that is at least partially horizontal. The cell bag rotator comprises a plurality of plates to which a respective cell bag is attached. The cell bag rotator is suitable for mixing cells with particles for performing various types of subsequent cell therapy processes on the cells.

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 provides compositions and methods for treating cancer in a human. The invention relates to targeting the stromal cell population in a tumor microenvironment. For example, in one embodiment, the invention provides a composition that is targeted to fibroblast activation protein (FAP). The invention includes a chimeric antigen receptor (CAR) which comprises an anti-FAP domain, a transmembrane domain, and a CD3zeta signaling domain.

Abstract: Devices and methods for affixing reinforcing material to a fascial incision in an abdominal wall to reinforce and augment closures thereof. The device includes first and second arms, each having a proximal end and a distal end extending away from a housing with a length therebetween. The distal end of the first arm is spaced from the distal end of the second arm such that the first and second arms are engageable with reinforcing material on opposing sides of a fascial incision. One or more fixation elements are deployable from the distal end of at least one of the first and second arms to affix the reinforcing material on opposing sides of the fascial incision.

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: 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: Methods for colloidal particle manipulation mediated by an elastic fluid responsive to changes in boundary conditions, including methods of controlling motion of colloidal particles using wavy wall boundary conditions. Methods for driving transitions in topological defect configurations of colloidal particles using wavy wall boundary conditions.

Abstract: Systems and methods for passive multi-directional illumination in light-sheet fluorescence imaging and microscopy are disclosed herein. An elliptical light shaping diffuser is placed in the illumination path between the source of a light-sheet and the illuminated sample. The light-sheet is diffused anisotropically along two directions perpendicular to its propagation direction, eliminating stripe artifacts in obtained images. The method includes converting a light-sheet into an elliptically diffuse light-sheet by passing it through an elliptical light shaping diffuser, illuminating a sample with the elliptically diffuse light-sheet. The system includes a light-sheet source, an elliptical light shaping diffuser adapted to convert the light-sheet into an elliptically diffuse light-sheet to illuminate the sample, typical microscopy optics and lenses, and image capturing elements.

Abstract: Inhibition of human epidermal growth factor receptor 2, human epidermal growth factor receptor 3, or of the heterodimerization of these proteins has presently been found to mitigate mechanically-induced lung injury or a lung-stretch injury. Provided are methods for treating a mechanically-induced lung injury or a lung-stretch injury, for increasing lung compliance, moderating alveolar epithelial permeability, or both in a subject having a mechanically-induced lung injury or a lung-stretch injury, for protecting a subject against a decrease in lung compliance, an increase in alveolar epithelial permeability, or both, as a result of a mechanically-induced lung injury or a lung-stretch injury, such methods involving the administration of an inhibitor of human epidermal growth factor receptor 2 (HER2) or human epidermal growth factor receptor 3 (HER3), or an inhibitor of the heterodimerization of HER2/HER3.

Abstract: Methods and systems are described for determining quantities of interest for particle therapy. An example method can comprise determining one or more first functions indicative of spectral fluence associated with a particle therapy. The one or more first functions can be based on a plurality of simulations of a particle beam. The method can comprise determining, based on the one or more first functions and a second function associated with calculating a quantity, data indicative of the quantity. The method can comprise causing output of the data indicative of the quantity.

Abstract: A method for non-invasive assessment of photoreceptor function in a mammalian subject comprises exposing a subject's eye to a visible light stimulus to initiate an intrinsic reflectance response in one or a population of photoreceptors and capturing multiple images of photoreceptor's intrinsic reflectance response to the stimulus. Patterns of variability in the intrinsic reflectance response of a single photoreceptor or population of photoreceptors are useful in diagnosis and treatment monitoring of an ocular condition, disease, disorder or a response to treatment for said ocular condition, disease or disorder.

Abstract: Disclosed are systems, methods, and devices for percutaneous retrieval of objects, such as endovascular devices, from an internal body space. The present inventions have vascular, non-vascular (gastrointestinal), and surgical (laproscopic) applications. The inventions include a retrieval end having one or more compressed states and an expanded state, the retrieval end adopting the expanded state when the retrieval end is deployed into a subject's internal body space, the retrieval end having a mouth through which an object can be passed into an interior space within the retrieval end when the retrieval end is in the expanded state, the mouth being adjustable between an opened state and a closed state; wherein when at least a portion of the retrieval end of the inner sheath is transitioned to a compressed state following capture of the object, the retrieval end exerts an inward force that at least partially collapses or compresses the object.

Abstract: A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.

Abstract: Mesogenic compounds having a structure of Formula (I), (II) or (III): where A, B, X1, L, TG, m, and n are defined as in claim 1. Compositions containing these compounds, articles made from these polymerized and prepolymerized compositions, and methods of estimating the elastic constants and anchoring constants of a liquid crystal materials and mapping topological defect structures in liquid crystals using these compounds.

Abstract: Compositions and methods for carboxymethylation of cytosine containing DNA and applications thereof for direct sequencing of 5mC are disclosed.

Abstract: The present invention includes compositions and methods for treating T cell lymphomas and leukemias. In certain aspects, the compositions and methods include CAR T cells targeting CD2, CD5, or CD7 and modified cells wherein CD2, CD5, or CD7 has been knocked-out.

Abstract: Provided herein are methods for identifying and treating BAV disease and/or aortopathy in a subject, and methods of improving outcome in a subject. The subject may be asymptomatic of BAV disease and/or aortopathy, experiencing symptoms of BAV disease and/or aortopathy, have BAV disease and/or aortopathy, or be a blood relative of an individual having BAV disease and/or aortopathy. Levels of sRAGE in the subject's biological sample are determined, compared to a control biological sample, and used as an indicator for the presence and severity of BAV disease and/or aortopathies, a tool to screen family members, and an indicator of the proper surgical or treatment regimens.

Abstract: The present disclosure features the use of chimeric CD3 proteins to modulate T cell Receptor (TCR) signaling. Specifically, the disclosure is based, in part, on the discovery that chimeric CD3 proteins (e.g., CD3delta, CD3gamma, and CD3epsilon) having all or most of their extracellular domain fused to an antigen binding domain can activate the TCR in the presence of a cognate antigen. The disclosure is further based on the observation that the above chimeric proteins can be potentiated through the inclusion of a co-stimulatory domain in the intracellular portion of the chimeric molecule. Thus, the preferred elements of the engineered signaling complexes of the disclosure include an antigen binding domain, an extracellular domain derived from one of the above CD3 proteins, and an intracellular co-stimulatory domain.