Abstract: In some aspects, the disclosure provides recombinant AAV and nucleic acid constructs having novel inverted terminal repeats (ITRs), cap, and/or rep genes. In some aspects, the disclosure relates to gene transfer methods using rAAVs described herein.

Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

Abstract: This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.

Abstract: A method for electrochemical treatment of water is provided. The method includes providing a flow-through reactor including a cathode and an anode, wherein the anode includes about 80 weight percent or greater of a sub-stoichiometric titanium oxide. The method further includes applying power to the cathode and the anode, passing a solution including water and a metal chloride through the flow-through reactor, and withdrawing the purified water.

Abstract: Provided herein are methods and compositions for disrupting expression of nuclear receptor interacting protein 1 (Nrip1) in adipose cells, and methods of use of such adipose cells for treating, or reducing risk of, a condition associated with an elevated body mass index (BMI).

Abstract: Aspects of the disclosure relate to compositions and methods for gene editing in a cell or subject. In some aspect, the present disclosure provides an isolated nucleic acid comprising an expression construct comprising transgene encoding a gene product flanked by a 5? homology arm and a 3? homology arm, wherein the expression construct is flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs).

Abstract: Aspects of the disclosure relate to methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof by administering an rAAV expressing one or more Ganglioside GM3 synthase (GM3S) isoforms via intracerebroventricular (ICV) administration.

Abstract: Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.

Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

Abstract: Aspects of the disclosure relate—compositions and methods useful for delivering minigenes—a subject. Accordingly, the disclosure is based, in part, on isolated nucleic acids and gene therapy vectors, such as viral (e.g., rAAV) vectors, comprising one or more gene fragments encoding a therapeutic gene product, such as a protein or peptide (e.g., a minigene). In some embodiments, the disclosure relates to gene therapy vectors encoding a ABCA4 protein (e.g., the gene product of ABCA4 gene) or a portion thereof. In some embodiments, compositions described by the disclosure are useful for treating diseases associated with mutations in the ABCA4 gene, for example, Stargardt disease.

Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, and/or BCKDHB, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).

Abstract: Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., an anti-inflammatory transgene encoding one or more gene products) to a joint. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for joint tissues (e.g., cartilage, skin, muscle, and bone cells). Methods of treating arthritis in a joint by administering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.

Abstract: A first image sensor device is operable to produce first orientation information indicating an orientation of the first image sensor device at a time of capturing a first image of an object. A second image sensor device is operable to produce second orientation information indicating an orientation of the second image sensor device at a time of capturing a second image of the object. The image capturing system further includes measurement hardware to measure a distance between the first image sensor device and the second image sensor device at times of capturing the first image and the second image. According to one configuration, a combination of the first orientation information, the second orientation information, and the distance between the first image sensor device and the second image sensor device is used to derive a 3-D stereo view of the object using the first image and the second image.

Abstract: A lightweight, efficient, autonomous sub-surface water data profiler (10) that can be easily programmed and deployed to collect sample data at a variety of water depths is disclosed. The profiler is a unique, simple-to-use, low-cost design with a low-cost depth control system that allows several dive/surface trips, an Android graphical interface for Bluetooth programming and a deployable antenna for retrieval after completing its mission. The collected data is achieved using an open-source Android interface and Bluetooth programming using Android and longer-range radio communications with a deployable antenna. The profiler is usable by anyone with a deep pond, lake, ocean or even a pool that they would like to monitor.

Abstract: A first distinct vehicle operational scenario is identified for an autonomous vehicle (AV). A first set of candidate vehicle control actions are received from a model that provides a first solution to the first distinct vehicle operational scenario. An action is selected from the first set of candidate vehicle control actions. The AV is controlled based on the action. The first solution is obtained offline in a first idealized situation that is decoupled from a current context of the AV.

Abstract: The present disclosure provides compositions and methods related to the modulation of the inflammation response activated by a host's immune system. In particular, the present disclosure provides novel inhibitors of proteins that exhibit pro-inflammatory activity (e.g., RIPK1, RIPK3, ZBP1, TRIF, CUL1, SKP1, and NF-?B, among others), as well as corresponding therapeutic compositions and methods of treatment using these inhibitors.

Abstract: Lane-level route planning includes obtaining lane-level information of a road, where the road includes a first lane and a second lane and the lane-level information includes first lane information related to the first lane and second lane information related to the second lane; converting the lane-level information to probabilities for a state transition function; receiving a destination; and obtaining a policy as a solution to a model that uses the state transition function.

Abstract: Aspects of the disclosure relate to recombinant gene editing complexes comprising a recombinant gene editing protein and guide RNA (gRNA) that specifically hybridizes to a region of a C90RF72 gene (e.g., a region flanking a G4C2 repeat or within a exonic region of the gene).

Abstract: The disclosure provides novel methods and compositions for gene editing. In particular, the disclosure relates to compositions and methods of making modified nucleic acid donor templates for highly efficient and precise gene editing.

Abstract: A gas sensor includes a biomaterial comprising electrically-conductive protein nanowires and at least two electrodes. The at least two electrodes are in operative arrangement with the protein nanowires and configured to provide a signal indicative of a change in conductivity of the protein nanowires. The conductivity of the protein nanowires is responsive to a change in concentration of a gas exposed to the biomaterial, such as ammonia, or to a change in relative humidity.