Abstract: Disclosed herein are materials and methods for the treatment of stress-related disorders and cancer. The disclosure provides an antibody, or antigen binding fragment thereof, that specifically binds to a region of corticotropin-releasing hormone (CRH). The disclosure also provides methods of treating a disorder associated with HPA axis activation, such as a stress-related disorder or cancer, comprising administering to a subject in need thereof an antibody or antigen binding fragment thereof described herein in an amount effective to treat the disorder.

Abstract: The invention relates to proteins that contain the CD4 domain 1 and the CD4 domain 2 (CD4 D1D2), wherein the CD4 D1D2 contains one or more mutations compared to wild-type human CD4 D1D2, and to methods of using the proteins for treating a human immunodeficiency virus (HIV) infection in a subject.

Abstract: Disclosed herein are recombinant AAV variant (e.g., variant serotype 5 (AAV5)) capsid proteins and variant capsid protein-containing viral particles with enhanced ability to transduce adult stem cells and neurons. Viral particles containing these capsid variants are capable of enhanced transduction of mammalian mesenchymal stem cells and neurons. The recombinant AAV5 variant proteins and viral particles disclosed herein were identified from a variant AAV5 capsid library that was engineered by making substitutions in variable regions of the capsid. Compositions of these variant AAV5 particles are provided that are useful for transducing and delivering therapeutic transgenes to cells, such as mesenchymal stem cells, and thus treat diseases and disorders pertaining to these cells.

Abstract: The subject invention provides therapeutic compositions, and uses thereof for the treatment or amelioration of injury to small intestine mucosa. In preferred embodiments, the composition comprises one or more nutrients and/or electrolytes that acquire or retain considerable absorptive capacity.

Abstract: An apparatus and method for the delivery and deposition of particles air-liquid-interface (ALI) cell cultures includes a sample inlet coupled to a growth tube having interior walls that are wet. The growth tube is configured to operate at a first temperature along a first length of the tube and a second temperature along a second length positioned between the first length and a growth tube outlet. The apparatus also includes a nozzle plate having a plurality of nozzles. The exposure chamber adapted to hold cell cultures at an air-liquid interface positioned underneath the plurality of nozzles and a temperature regulator adapted to control a temperature of the exposure chamber. The apparatus also includes a controller including instructions operable to cause the controller to maintain a relative humidity within the exposure chamber by controlling at least the second temperature of the growth tube and the temperature of the exposure chamber.

Abstract: Methods and systems are directed to protect a target logic circuit against optical probing attacks and conceal a logic state in the target logic circuit. The method comprises designing a concealing logic circuit. The concealing logic circuit can be coupled to complement of input signals of the target logic circuit, inserted as neighbor logic circuit of the target logic circuit, and placed in close optical proximity to the target logic circuit. The concealing logic circuit can operate in the opposite operation mode or logic state of the target logic circuit in response to the complement of the input signals to the target logic circuit. The method can further comprise designing an evaluation circuit that generates the complement of the input signals and minimizes a path delay between the target logic circuit input and the concealing logic circuit input.

Abstract: Provided herein are compounds (e.g., compounds of Formula (I)), their mechanism of action, and methods of treating diseases and disorders (e.g., cancer) using the compounds provided herein (e.g., compounds of Formula (I)). Also disclosed herein are N methods of inhibiting and degrading kinases (e.g., CDK9, CDK10, or anaplastic lymphoma kinase).

Abstract: Described are alcohol-induced liver cancer model mice, methods of generating the alcohol-induced liver cancer model mice, and methods of using the alcohol-induced liver cancer model mice. Methods of treating alcoholic liver disease, including hepatocellular carcinoma, are also described.

Abstract: Provided herein are amino acid compositions useful for increasing the translocation of the cystic fibrosis transmembrane conductance (CFTR) protein from the cytoplasm to the plasma membrane, particularly in epithelial cells. Methods for increasing the concentration of CFTR in the plasma membrane, increasing chloride ion transport, and increasing water transport are also provided. These compositions and methods are useful in treating cystic fibrosis in subjects bearing one or more mutations in the CFTR protein. Use of these compositions for the treatment of cystic fibrosis and in the preparation of a medicament for the treatment of cystic fibrosis are also encompassed herein.

Abstract: The present disclosure provides methods, uses, kits, and compositions comprising a compound of Formula (I), and either a second compound of Formula (I) or a compound of Formula (II). In one aspect, the methods are for treating a neurological disease associated with repeat expansions and/or RAN protein accumulation, reducing the level of one or more repeat associated non-AUG (RAN) proteins, and reducing the accumulation of RAN proteins in a subject and/or biological sample. Exemplary diseases associated with repeat expansions include, but are not limited to, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, myotonic dystrophy type 1, myotonic dystrophy type 2, spinocerebellar ataxia, Alzheimer's disease, Huntington's disease, Fragile X Tremor Ataxia Syndrome, and Fragile XE syndrome.

Abstract: In several embodiments, the present disclosure relates to nucleic acids, compositions, and methods for the delivery of a therapeutic gene to a subject. In several embodiments, the therapeutic gene is through the use of a viral vector. In several embodiments, the viral vector is an adeno-associated virus. In several embodiments, the therapeutic gene is delivered to treat a cardiac disease, injury or other disorder.

Abstract: In several embodiments, the present disclosure relates to nucleic acids, compositions, and methods for the delivery of a therapeutic gene to a subject. In several embodiments, the therapeutic gene is through the use of a viral vector. In several embodiments, the viral vector is an adeno-associated virus. In several embodiments, the therapeutic gene is delivered to treat a cardiac disease, injury or other disorder.

Abstract: Provided herein is a method, apparatus, and system for teleoperated, functional electric stimulation actuated rehabilitative cycling. Methods may include: receiving, at a master system, rotational input generating master system sensor input having a master system cadence; providing the master system sensor input including the master system cadence to a controller based on the rotational input from a rehabilitation participant or a remote therapist received at the master system; receiving, at a slave system, rotational input from the rehabilitation participant generating slave sensor input having a slave system cadence; providing the slave system sensor input including the slave system cadence to the controller based on the rotational input from the participant received at the slave system; and providing, from the controller, feedback through the master system in response to a difference between the master system cadence and the slave system cadence.

Abstract: The present invention provides methods and compositions for optimally co-expressing in a primate subject a tyrosylprotein sulfotransferase (TPST) and a lentiviral gp120-binding molecule to provide potent and long term protection against lentiviral infections.

Abstract: Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering RNA to a subject in order to reduce expression of one or both alleles of an endogenous RHO gene (for example a mutant rho allele associated with retinitis pigmentosa) in a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.

Abstract: Various embodiments provide methods, systems, computer program products, apparatuses, and/or the like for assessing vulnerability of an IC design to fault injection attacks, such as through a security property-driven vulnerability assessment framework for efficiently evaluating faults with respect to certain security properties associated with the IC design. In one embodiment, a method is provided. The method includes generating, using a fault-injection technique specification, one or more fault models describing attributes of one or more faults. The method further includes selecting, using the fault models and executable security properties associated with a design file of an IC design, a fault list identifying a plurality of possible faults for the IC design. The method further includes identifying, based at least in part on performing a fault simulation on the design file with the fault list, critical locations of the IC design.

Abstract: Methods for producing a custom-made stapes prosthesis for a patient and stapes prostheses so produced. The methods may include obtaining a three-dimensional image of a natural stapes bone of the patient; and forming a stapes prosthesis that closely approximates a three-dimensional shape of the natural stapes bone via an additive manufacturing technique based on the three-dimensional image. The stapes prosthesis comprises a suitable material that is not osteogenic and is not cytotoxic. The stapes prosthesis may include a plurality of pores having an average size of from about 5 to about 100 micrometers and may exhibit a frequency of sound transmission in a range of from about 2 to about 30 kilohertz.

Abstract: The invention provides antibodies, antibody-based fragments or antibody fragments (antigen-binding fragments), as well as derivative molecules such as bispecific T-cell engagers, antibody-drug conjugates (ADCs) and chimeric antigen receptors (CARs), that specifically recognize Siglec-6 and related compositions. Also provided in the invention are methods of using such antibodies in various diagnostic and therapeutic applications.

Abstract: Provided herein are methods and compositions for expressing Otoferlin, e.g., utilizing adeno-associated viral (AAV) particles. Further provided herein are compositions of AAV particles comprising one or more polynucleotides encoding Otoferlin are codon optimized for expression in human cells. Such methods and compositions may be useful for treatment of diseases and disorders such as Deafness, Autosomal Recessive 9 (DFNB9). Also provided are kits comprising such compositions.

Abstract: The invention pertains to the use of VirB10 to immunize a host against an infection by a bacterium having T4SS. The invention provides a vaccine comprising VirB10, a fragment of VirB10, a polynucleotide encoding VirB10 or a polynucleotide encoding a fragment of VirB10 and a pharmaceutically acceptable carrier and/or adjuvant. The invention also provides a method of immunizing a host against an infection caused by a bacterium having T4SS, the method comprising administering to the host a vaccine of the invention. The vaccines and the methods of the invention can be used to immunize against infections caused by bacteria having T4SS in dogs, rabbits, cats, pigs, cattle, sheep, goats, deer, horses, rodents and humans.