Abstract: Methods and uses for diagnosing and treating Tourette syndrome are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.

Abstract: Compositions and methods for the detection and treatment of ADHD are provided.

Abstract: The disclosure is directed to crystalline forms and mixtures thereof of R-fasoracetam. Such crystalline forms include Form II monohydrate R-fasoracetam and the anhydrate form of R-fasoracetam. The disclosure further includes mixtures of the anhydrate form of R-fasoracetam together with one or more of Form I monohydrate R-fasoracetam and Form II monohydrate R-fasoracetam.

Abstract: This application relates to methods of treating attention deficit hyperactivity disorder (ADHD), 22q deletion and/or duplication syndrome, and co-morbidities with a nonselective activator of metabotropic glutamate receptors, such as fasoracetam, for example, in subjects having a genetic alteration in at least one metabotropic glutamate receptor (mGluR) network gene.

Abstract: Disclosed are devices and methods directed to oxygenators for use in extracorporeal circuits for supporting a fetus. An oxygenator for use with an extracorporeal circuit includes a housing defining a cavity therein and a gas exchanger disposed within the interior cavity. The cavity is configured to receive blood a fetus. The gas exchanger is configured to receive a sweep gas and further configured to contact the blood within the cavity, such that at least oxygen gas and carbon dioxide gas is permitted to diffuse between the blood and the gas exchanger.

Abstract: The present invention provides methods, agents, compounds, and compositions useful for administering to subjects having or at risk of having anemias. In certain embodiments, methods herein comprise administering two agents to a subject, which are useful for the treatment of an anemia.

Abstract: A portable pupillometer for evaluating one or more pupils of a subject includes an optical assembly to stimulate the one or more pupils. The optical assembly has a light source to deliver a stimulus to the one or more pupils and a detector to capture images of the one or more pupils during delivery of the stimulus. A control device is coupled to the optical assembly, which has a controller to perform a plurality of operations in accordance with instructions stored in a digital memory. The plurality of operations includes: obtaining pupillary light reflex data comprising a plurality of Pupillary Light Reflex metrics during stimulation of the one or more pupils; analyzing the pupillary light reflex data based on one or more characteristics of the subject; determining a probability of a concussion injury suffered by the subject; and outputting the probability to a user of the pupillometer.

Abstract: Factor VIII variants and methods of use thereof are disclosed.

Abstract: Methods and compositions for producing hemoglobin and treating alpha-thalassemia are disclosed.

Abstract: Expression vectors for rapid and high-throughput cloning, expression and screening of chimeric antigen receptor (CAR) constructs are provided.

Abstract: Compositions and methods for effecting base editing to correct mutations in the phenylalanine hydroxylase gene, thereby curing phenylketonuria, are disclosed.

Abstract: A method for treating scoliosis in a patient is disclosed. The method comprises identifying a spinal column having a lateral convex curve; providing a first anchor, a second anchor, and a tether; securing the first anchor to a posterior region of a first vertebra; securing the second anchor to a posterior region of a second vertebra; securing a first tether end at a first tether connection location; and securing a second tether end at a second tether connection location. A scoliosis treatment system comprises anchors, each having a tether connection location positioned at a first location offset in a first lateral direction from a midsagittal plane of the first or second vertebra, and the anchors do not include any tether connection locations offset in in a second lateral direction, opposite the first lateral direction, from the midsagittal plane of the first vertebra or second vertebra.

Abstract: Provided herein are compositions and methods for providing long-term and sustained CAR T cell efficacy. For example, expression cassettes that encode both a GPC2-targeting chimeric antigen receptor and a bispecific innate immune cell engager binding both GD2 on tumor cells and CD16A on natural killers (NK) and macrophages are provided. In addition, T cells that both expression a GPC2-targeting chimeric antigen receptor on their surface and secrete a bispecific innate immune cell engager binding both GD2 on tumor cells and CD16A on natural killers (NK) and macrophages are provided.

Abstract: Provided herein are systems for increasing AAV particle production. These systems comprise producer cell lines adapted for the production of AAV particles, as well as methods of producing AAV particles using said producer cell lines. Also provided are AAV particles produced by said production systems, producer cell lines and methods.

Abstract: This disclosure is directed to methods of treating or inhibiting mitochondrial dysfunction or mitochondrial disease in a subject comprising administration of and effective amount of one or more of (+) Epicatechin (?) epicatechin, 11-hydroxyprogesterone, and 11-?-hydroxypregnenolone and other efficacious agents in a pharmaceutically acceptable carrier. Agents can be administered alone or in certain synergistic combinations. The compositions and methods described effectively reduce or alleviate primary respiratory chain dysfunction symptoms, and have efficacy for improving cellular resiliency, stress resistance, and symptoms associated with primary, secondary, or acute respiratory chain dysfunction.

Abstract: A cerebral monitoring device for determining failure of a shunt used to treat pediatric hydrocephalus. The cerebral monitoring device including a controller configured to control an optical instrument to emit multi-spectral light to illuminate a cranial tissue of the patient, and control an optical detector to detect multi-spectral light emitted from the illuminated cranial tissue of the patient. The controller also configured to compare the emitted multi-spectral light to the detected multi-spectral light, compute cerebral blood flow (CBF) data based on the comparison, compute a pulsatility index of the CBF data, compute a pulsatility index of blood pressure of the patient, compute intracranial pressure (ICP) based on the pulsatility index of the CBF data and the pulsatility index of the blood pressure, and determine shunt failure based on blood oxygen saturation of the patient and the ICP of the patient.

Abstract: A power tool, such as a cast saw is disclosed. The cast saw is equipped with at least one thermocouple and and/or at least one infrared detector that measure the temperature of the cutting blade on the cast saw or the area in the cast immediately surrounding the cutting blade. The cast saw is also equipped with at least one LED that can alert a user of the cast saw when blade or the area in the cast immediately surrounding the blade is approaching or has reached a temperature that may burn a patient. Also disclosed is a shroud for the cutting blade of the cast saw. The shroud not only surrounds at least part of the blade but also houses the thermocouple or infrared detector and the LEDs(s). A method of operating the cast saw to cut a cast off of a patient is also disclosed.

Abstract: Compositions and methods for the detection and treatment of ADHD are provided.

Abstract: The neuroblastoma immunopeptidome is enriched with peptides derived from proteins essential for tumorigenesis including the unmutated peptide QYNPIRTTF (SEQ ID NO: 1) discovered on HLA-A*24:02 which is derived from the neuroblastoma dependency gene and master transcriptional regulator PHOX2B. To target QYNPIRTTF, peptide-centric chimeric antigen receptors (PC-CARs) were developed via a counter panning strategy using predicted potentially cross-reactive peptides. Informed by computational modeling, PHOX2B peptide¬ centric CARs were demonstrated to also recognize QYNPIRTTF (SEQ ID NO: 1) presented by HLA-A*23:01 and the highly divergent HLA-B*14:02. Potent and specific killing of neuroblastoma cells expressing these HEAs in vitro was shown along with complete tumor regression in mice.

Abstract: Provided herein are engineered red blood cells expressing rare blood antigen group profiles, and methods of making use the same, are described. Also provided are recombinant reagent red blood cells that express or lack the expression of at least one protein (e.g., a blood group antigen) on its surface and uses thereof.