Abstract: The present invention provides methods and compositions for the treatment of mucopolysaccharidoses (MPS).

Abstract: Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such recombinant meganucleases in combination with mitochondrial transit peptides in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells wherein the mtDNA has been modified or edited.

Abstract: Provided herein are compositions and methods of use thereof for the treatment of metabolic disease such as diabetes and obesity. One embodiment provides a method of treating a metabolic disease or syndrome in a subject in need thereof by administering to the subject a therapeutically effective amount of a growth hormone releasing hormone (GHRH) antagonist or a pharmaceutical composition comprising the GHRH antagonist to reduce triglyceride-rich-lipoproteins (TRL) in the subject to treat the metabolic condition or syndrome.

Abstract: A system measures performance activity of a user using a motion capture device, such as one or more knee sleeves each a plurality of inertial-measurement unit. Captured motion characteristic data is classified against previously captured data and then mapped to particular audio effects, which are then altered in different ways depending on the classification and provided the user. The audio feedback works to improve the user's performance of the activity by changing between negative and positive feedback depending on the user's performance. The system regularly measures the motion capture data, altering audio effects provided to the user, until the user reaches an improvement goal.

Abstract: Described herein are materials and methods for the treatment of prostate cancer.

Abstract: The present invention provides a method of treating heart failure with reduced ejection fraction, by administering to a patient at risk of such damage, a pharmaceutically effective amount of a composition which inhibits the anchoring of PP2A to mAKAP?. This composition is preferably in the form of a viral based gene therapy vector that encodes a fragment of mAKAP? to which PP2A binds.

Abstract: Activation of STimulator of INterferon Genes (STING) triggers cytokine production and facilitates tumor antigen cross-presentation. In an embodiment of the present invention, STING-dependent innate immune signaling pathway activators (STAVs) can be delivered to antigen presenting cells. In various embodiments of the present invention, the STAVs can be delivered in lipid nanoparticle formulations. In various embodiments of the present invention, the range of cancers amenable to STAV therapy can be extended using a non-cell-based nanoparticle strategy that effectively delivers Nano-STAVs into the Tumor Micro Environment (TME) to potently generate anti-tumor cytotoxic T cell activity. The STAV formulations can be introduced into solid tumors present in the mammal. Alternatively, the Nano-STAVs can be introduced through direct inoculation. The lipid nanoparticles stick to the tumor cells and are co-phagocytosed to activate STING in APC's.

Abstract: Provided herein are compounds useful in treating a central nervous system disorder associated with neuronal and/or axonal damage, methods for their preparation, and related pharmaceutical compositions. For example provided herein are compounds of Formula (I): and pharmaceutically acceptable salts and compositions including the same.

Abstract: Systems, methods, and computer readable media for determining cognitive impairment (CI) in patients are provided herein. Various regional structural-functional parameters of the retina can serve as biomarkers for the detection of CI. The method can include forming a database including a quantification of retinal structure and retinal function of a plurality of eyes associated with a plurality of patients, providing a baseline cognitive impairment (CI) reference. The method can include determining a measure of functionality of neurons in the retina based on an electroretinogram (ERG) of a patient. The method can include determining a structural measure of the first retina based on a generalized dimension spectrum and singularity spectrum of the skeletonized retinal image, and a lacunarity parameter of the skeletonized retinal image. The method can include determining a level of cognitive impairment based on the structural and functional measures.

Abstract: The compositions and methods described herein include methods and agents that inhibit inflammasome signaling in a mammal such as antibodies directed against inflammasome components used alone or in combination with extracellular vesicle uptake inhibitor(s) or other agents. Also described herein are compositions and methods of use thereof for detecting and treating early-stage Alzheimer's disease as well as other inflammatory neurologic conditions.

Abstract: A device and method for aspirating graft tissue and delivering the graft tissue to a target delivery site (for example, when performing Descemet's membrane endothelial keratoplasty). In one embodiment, an injector comprises a cylinder and a plunger at least partially located within the cylinder, the plunger being rotatably advanceable and retractable within the cylinder. In one aspect of the embodiment, rotating the plunger in a first direction within the cylinder controllably aspirates a graft tissue into the injector and rotating the plunger in a second direction opposite the first direction controllably ejects the graft tissue from the injector.

Abstract: The disclosure provides a method of treating a polyamine imbalance-related disorder. The method comprises administering phenylbutyrate to a subject in need thereof, thereby treating the polyamine imbalance-related disorder.

Abstract: The present invention provides a method of treating heart failure with reduced ejection fraction, by administering to a patient at risk of such damage, a pharmaceutically effective amount of a composition which inhibits the anchoring of PP2A to mAKAP?. This composition is preferably in the form of a viral based gene therapy vector that encodes a fragment of mAKAP? to which PP2A binds.

Abstract: Computer-implemented processes for precision therapeutic biomarker screening for cancer include using a model-based overlapping clustering framework to assess large numbers of possible drugs and drug combinations against patient data, including cell line responsiveness. A multivariate regression model has been developed, along with a latent overlapping cluster indicator variable. The techniques employ a new finite mixture of multivariate regression (FMMR) model and expectation-maximization (EM) algorithm for modeling. The techniques can analyze large amounts of drug data and identify complex overlapping drug clusters, as well as cluster-wise drivers that facilitate identification of new drugs for treating pathologies, such as cancer, in patients.

Abstract: Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such recombinant meganucleases in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells wherein the mtDNA has been having modified or edited.

Abstract: Provided herein are compounds of the general Formula (I) which act as kinase inhibitors, e.g. ROCK, S6K, and/or PKC inhibitors, and are useful in neurite growth and axonal growth.

Abstract: Described herein are materials and methods for inducing therapeutic hypothermia in a subject in need thereof.

Abstract: The invention provides a method of treating or preventing pain in a subject in need thereof. The method comprising administering to the subject an expression vector comprising a nucleic acid sequence encoding carbonic anhydrase (10) or carbonic anhydrase (11) such that the nucleic acid is expressed to produce carbonic anhydrase (10) or carbonic anhydrase (11). Alternatively, the method comprising administering to the subject an expression vector comprising a nucleic acid sequence encoding a carbonic anhydrase (8) fragment such that the nucleic acid is expressed to produce the carbonic anhydrase (8) fragment.

Abstract: Described herein are materials and methods for stratifying risk for reactivation of a latent viral infection, such as CMV infection. The methods are particularly useful for immune compromised subjects. Also described herein are interventions, including therapeutic, prophylactic, and monitoring interventions, for subjects determined to be at elevated risk.

Abstract: Disclosed herein are recombinant meganucleases engineered to recognize and cleave a recognition sequence present in the human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such recombinant meganucleases in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells wherein the mtDNA has been having modified or edited.