Abstract: The present invention is inter alia directed to artificial nucleic acid constructs, preferably RNA, comprising at least one coding sequence encoding at least one transcription factor inhibitor for reducing or inhibiting the activity of a target transcription factor in a cell. A preferred transcription factor inhibitor according to the invention is a Runt-related transcription factor (RUNX) inhibitor, for example a RUNX trap comprising at least one amino acid sequence for binding a RUNX transcription factor and at least one amino acid sequence for capturing or trapping RUNX. Further provided are pharmaceutical compositions comprising the artificial nucleic acid, preferably formulated in polyethylene glycol/peptide polymers, polymeric carriers, or lipid-based carriers. Also provided are methods of treating or preventing disorders, diseases, or conditions, and medical uses.

Abstract: The present application relates to compositions and methods for eliciting an immune response in a subject using an Adeno-Associated Virus (AAV) AAV11 vector comprising an AAV11 capsid protein and a nucleic acid encoding a transgene operably linked to a promoter, wherein the transgene encodes an immunogenic polypeptide. Further disclosed are immunogenic polypeptides that are used for the compositions and methods.

Abstract: The invention provides compositions and methods for treating optic neuropathic disorders.

Abstract: Methods are described for predicting ancestral sequences for viruses or portions thereof. Also described are predicted ancestral sequences for adeno-associated virus (AAV) capsid polypeptides. The disclosure also provides methods of gene transfer and methods of vaccinating subjects by administering a target antigen operably linked to the AAV capsid polypeptides.

Abstract: Methods, systems, and devices are provided for measuring eye refraction without a lens, and more particularly, for measuring eye refraction with a mobile device application. An exemplary method includes measuring a distance between a patient and a mobile device, presenting to the patient one or more visual targets sized and shaped to represent perfect vision such as by using Vernier targets or grating targets, instructing the patient to indicate whether the patient can accurately read the visual targets and, if not, to move closer to the mobile device until the patient can accurately read the visual targets, calculating a vision prescription for the patient based on the visual targets and a final distance between the patient and the mobile device, and displaying the vision prescription for the patient.

Abstract: Described herein are polyhedrin-based slow release growth factor systems and methods of use thereof for neuroprotection of host and donor retinal ganglion cell neurons.

Abstract: Methods, systems, and devices are provided for assessing visual function, especially in animals. For example, a device can be used for assessing visual functions, such as PLR and OMR, in animals that includes a visual stimulus unit. The device can also include one or more input devices that include at least one camera that is configured to monitor movement of the test subject and a processor that is configured to analyze the movement of the test subject taken by the camera and to assess visual functions of the test subject. The device can be used in a system including a designated general location for the test subject in which the test subject can see the visual stimulus unit and move freely while being monitored by the camera.

Abstract: Methods and compositions for modulating the transduction efficiency of an adeno-associated vims (AAV) into a cell or tissue are provided.

Abstract: The present subject matter provides, inter alia, compositions, systems, kits, and methods for diagnosing and treating small vessel diseases (SVDs).

Abstract: The present application relates to compositions and methods for eliciting an immune response in a subject using an AAV11 vector comprising an AAV11 capsid protein and a nucleic acid encoding a transgene operably linked to a promoter, wherein the transgene encodes an immunogenic polypeptide.

Abstract: Methods, systems, and devices are provided for measuring dark adaptation of one or both eyes of a patient, and more particularly, for measuring dark adaptation with a mobile device application. An exemplary method includes exposing an eye of a patient to a light source to bleach a retinal location of the eye, displaying on a mobile device a figure with a luminance and waiting until the patient communicates with the mobile device to acknowledge that the patient can see the figure, measuring and recording a level of the luminance and a time period between first displaying the figure and the patient communicating with the mobile device, continuing to display additional figures with decreasing luminance one at a time, and determining by a processor dark adaptation measurements of the patent based on the measured and recorded luminance and time periods.

Abstract: Methods are described for predicting ancestral sequences for viruses or portions thereof. Also described are predicted ancestral sequences for adeno-associated virus (AAV) capsid polypeptides. The disclosure also provides methods of gene transfer and methods of vaccinating subjects by administering a target antigen operably linked to the AAV capsid polypeptides.

Abstract: Provided herein are materials and methods for efficiently delivering nucleic acids to cochlear and vestibular cells.

Abstract: This disclosure describes compositions, methods, and systems for constructing defined variation in a contiguous functional genetic unit in association with a unique sequence identifier (“a barcode”) in a combinatorial manner.

Abstract: Methods, systems, and devices are provided for measuring eye refraction without a lens, and more particularly, for measuring eye refraction with a mobile device application. An exemplary method includes measuring a distance between a patient and a mobile device, presenting to the patient one or more visual targets sized and shaped to represent perfect vision such as by using Vernier targets or grating targets, instructing the patient to indicate whether the patient can accurately read the visual targets and, if not, to move closer to the mobile device until the patient can accurately read the visual targets, calculating a vision prescription for the patient based on the visual targets and a final distance between the patient and the mobile device, and displaying the vision prescription for the patient.

Abstract: This disclosure provides compositions and methods for altering or changing the tissue tropism, e.g., liver tropism, of adeno-associated viruses (AAV).

Abstract: Methods and compositions are provided that can be used to modify the assembly activating protein (AAP)-dependence of an adeno-associated virus (AAV).

Abstract: The present invention relates to substantially homogenous populations of human retinal progenitor cells having the following positive surface markers: SSEA4, CD73, PTK7 and PSA-NCAM. The invention also relates to method for preparing such substantially homogeneous cell populations from human tissue using cell sorting techniques.

Abstract: Methods, systems, and devices are provided for measuring dark adaptation of one or both eyes of a patient, and more particularly, for measuring dark adaptation with a mobile device application. An exemplary method includes exposing an eye of a patient to a light source to bleach a retinal location of the eye, displaying on a mobile device a figure with a luminance and waiting until the patient communicates with the mobile device to acknowledge that the patient can see the figure, measuring and recording a level of the luminance and a time period between first displaying the figure and the patient communicating with the mobile device, continuing to display additional figures with decreasing luminance one at a time, and determining by a processor dark adaptation measurements of the patent based on the measured and recorded luminance and time periods.

Abstract: Methods, systems, and devices are provided for measuring eye refraction without a lens, and more particularly, for measuring eye refraction with a mobile device application. An exemplary method includes measuring a distance between a patient and a mobile device, presenting to the patient one or more visual targets sized and shaped to represent perfect vision such as by using Vernier targets or grating targets, instructing the patient to indicate whether the patient can accurately read the visual targets and, if not, to move closer to the mobile device until the patient can accurately read the visual targets, calculating a vision prescription for the patient based on the visual targets and a final distance between the patient and the mobile device, and displaying the vision prescription for the patient.