Abstract: The present disclosure relates to compositions and methods for treating Williams syndrome (WS), herein identified as a neurodevelopmental oligodendrocyte hypomyelination-associated disease, and to compositions and methods for treatment of other neurodevelopmental myelination abnormality diseases or disorders.

Abstract: Disclosed herein are modified Clostridial Botulinum neurotoxin (BoNT) polypeptides with a modified receptor binding domain of Clostridial Botulinum serotype A1 or A2. Modifications include substitution amino acid mutations. Isolated modified receptor binding domains, chimeric molecules, pharmaceutical compositions, and methods of using the same are also disclosed.

Abstract: Described herein are techniques to aid clinicians and researchers in determining a condition of connective tissue as it relates to tissue development, growth and maturation, tissue remodeling and healing following injury, and risk of injury based on a magnetic resonance (MR) image of the tissue. Such techniques may be useful to clinicians by providing insights on factors that influence the growth and maturation of connective tissues as well as those that impact the risk of connective tissue injury and response to treatment. These insights can be used in a variety of ways, including to guide or develop patient specific risk assessment and prevention strategies, treatment plans, and postoperative care plans for individuals at risk of connective tissue injuries and those with injured connective tissues, such as an anterior cruciate ligament (ACL) injury.

Abstract: Antibodies, and antigen-binding fragments thereof, that specifically bind to Cluster of Differentiation 1a (CD1a) are provided. Embodiments include uses, and associated methods of using the antibodies, and antigen-binding fragments thereof.

Abstract: Provided herein are methods and products for detecting analytes in a sample. The analytes may be rare analytes such as biomarkers in a biological sample. These methods make use of nucleic acid nanoswitches that adopt a particular conformation and have a particular length in the presence of an analyte.

Abstract: Methods and apparatus for cleaning a central venous catheter port are disclosed. An apparatus includes a body, a coupling configured to connect the body to the hub, a cleaning cap coupled to the body, and an actuator disposed within the body for rotating and translating the cap relative to the hub. The cleaning cap includes a cap body defining a cavity and a cleaning member disposed within the cavity, the cleaning member having threads that engage with the threads on the hub.

Abstract: Provided herein are polypeptides that are truncated modified recombinant forms of ADAMTS13, nucleic acid molecules encoding the polypeptides, drug delivery compositions and pharmaceutical compositions comprising the polypeptides, and methods of using the polypeptides and compositions (e.g., in the treatment of thrombotic thrombocytopenic purpura (TTP)).

Abstract: An apparatus for measuring a characteristic of a sample using a centrifuge and optical components is disclosed. The centrifuge may be a standard benchtop centrifuge. The optical components may be sized and dimensioned to fit, along with the sample, inside the centrifuge.

Abstract: The present disclosure generally relates to systems and methods for multi-focal imaging, for example, for determining nucleic acids in cells or other samples. In some cases, multiple focal planes may simultaneously be determined, e.g., by using a plurality of detectors, such as a plurality of cameras, which image the same sample, but at least some of which are focused on different focal planes within the sample. Thus, the sample may be imaged in 3 dimensions, e.g., without sample refocusing. In certain cases, this may improve the resolution of imaging, in space and/or time. Various embodiments can be used to increase imaging throughput and/or resolution in image-based approaches, e.g., for single-cell molecular profiling such as multiplexed error robust fluorescence in situ hybridization (MERFISH), or for other applications.

Abstract: Therapeutic chondrisome compositions and related methods are described.

Abstract: Therapeutic chondrisome and mitoplast compositions and related methods are described.

Abstract: The disclosure provides fusion proteins comprising a pleckstrin homology (PH) domain and a variant of Botulinum neurotoxin E (BoNT E) protease that cleaves certain non-canonical protein targets (e.g., PTEN). Fusion proteins described in the disclosure are useful for cleaving target proteins found in a cell, that is, in an intracellular environment. Aspects of the disclosure provide methods for inhibiting PTEN amount, activity, or function in a cell or subject, the methods comprising administering to a call or subject a fusion protein described herein.

Abstract: A device and system for flushing a shunt catheter utilizes the available cerebrospinal fluid (CSF) to flush a blocked catheter. The CSF is pressurized to a predetermined amount and then allowed to suddenly, rapidly and forcefully purge any occlusions. The rapid release of CSF produces flow jets from the catheter pores into the ventricle. This impulse, or “cough”, will push and divert choroid plexus and/or other blockages away from the pores. The device and system may then be allowed to refill at a slow rate, thus reducing the possibility of rapid suction of fluid back into the system and the attendant possibility of drawing the choroid plexus back into the pores. The catheter at the proximal end may also include back-up pores that can be opened to restart flow from the ventricle should the primary pores remain blocked after a flushing attempt.

Abstract: Provided herein, in some aspects, are delivery vehicles comprising a ceramide and an agent to be delivered attached to the ceramide. In some embodiments, the ceramide does not comprise a fatty acid (i.e., is a sphingosine). In some embodiments, the ceramide comprises a fatty acid. In some embodiments, the ceramide is a glycoceramide. In some embodiments, the agent is attached to the ceramide covalently (e.g., via a linker). In some embodiments, the agent to be delivered is a therapeutic agent. The ceramide is able to deliver the agent to a cell or to a cellular compartment, as well as across the musical barrier. In some embodiments, agents delivered using the ceramide described herein exhibit longer half-life, compared to agents delivered alone. Methods of delivering a therapeutic agent to a subject for treating a disease using the ceramide delivery vehicle are also provided.

Abstract: A replacement heart valve device is disclosed. In some embodiments, the device includes a frame coupled to one or more leaflets that are moveable between open and closed configurations. In some embodiments, the frame comprises at least two frame sections that join at a pair of commissural posts. In some embodiments, the device may be geometrically accommodating to adapt to different vasculature shapes and sizes and/or to be able to change size while implanted within a growing patient.

Abstract: The disclosure provides modified biotin-binding proteins which can be expressed in soluble form in high yield in bacteria. Also provided are fusion proteins comprising the modified biotin-binding protein and an antigen. The disclosure further provides non-hemolytic variants of alpha-hemolysin from S. aureus and fusion protein comprising non-hemolytic variant of alpha-hemolysin and a biotin-binding domains. Immunogenic compositions comprising the proteins are also disclosed and use of such immunogenic compositions for inducing an immune response or for vaccinating a subject are also disclosed.

Abstract: As described below, the present invention features compositions and methods for neuroprotection and/or neuroregeneration of damaged or degenerating neurons. In various embodiments, the compositions and methods of the present disclosure are used to treat a neurodegenerative disease and/or nervous system injury. The methods in various embodiments include reducing or eliminating activity or expression of a target gene(s) and/or a polypeptide(s) expressed by a target gene(s) in a neuron.

Abstract: Described herein are methods and compositions for treating cancer. Aspects of the invention relate to administering to a subject having cancer an asparaginase and an agent that inhibits G6PD. Another aspect of the invention relates to administering an asparaginase to a subject having cancer that comprises a G6PD deficiency.

Abstract: The disclosed invention provides a method of treating a central nervous system (CNS) disease or disorder comprising administering to a subject diagnosed with the CNS disease or disorder a composition comprising a population of genetically modified, programmed cell death-1 receptor ligand (PD-L1)+-expressing hematopoietic stem cells (HSCs), wherein the CNS disease or disorder involves inflammation of the CNS. In one embodiment, the CNS disease or disorder is Multiple Sclerosis (MS). In certain aspects, the hematopoietic stem cells (HSCs) are obtained from a subject having a CNS disease or disorder prior to modification.

Abstract: Surgical simulation devices and their methods of use are described. In some embodiments, a surgical simulation device includes a base, a first surgical model configured to simulate a first cleft lip condition, and a second surgical model configured to simulate a second cleft lip condition. The device includes a model support configured to be releasably engaged with the base. The first surgical model is disposed on a first surface of the model support and the second surgical model is disposed on a second surface of the model support. The base includes an internal volume sized and shaped to receive one of the first surgical model and the second surgical model. The other of the first surgical model and the second surgical model is at least partially exposed when the model support is engaged with the base.