Abstract: The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.

Abstract: Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a cancer associated with DUX4 expression or overexpression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides microRNA (miRNA) for inhibiting or downregulating the expression of DUX4 and methods of using said miRNA to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression.

Abstract: The present disclosure relates to recombinant adeno-associated vims (rAAV) delivery of a neurotrophin 3 (NT-3) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for NT-3 gene therapy to improve muscle strength, stimulate muscle growth and to treat muscle wasting disorders, such as muscular dystrophy and Charcot-Marie-Tooth neuropathy.

Abstract: This disclosure provides isolated or recombinant polypeptides that are useful to vaccinate individuals suffering from chronic/recurrent biofilm disease or as a therapeutic for those with an existing infection. The individual's immune system will then naturally generate antibodies which prevent or clear these bacteria from the host by interfering with the construction and or maintenance of a functional protective biofilm. Alternatively, antibodies to the polypeptides can be administered to treat or prevent infection. Bacteria that are released from the biofilm by our technology are more readily cleared by the remainder of the host's immune system.

Abstract: The disclosure provides for methods of predicting disease progression comprising detecting a modification of one or more disease markers in a glial cell or neuronal cell generated from a skin cell of the subject. The disclosed methods include methods of identifying subjects that are responsive to a therapeutic agent and methods of determining effectiveness of a therapeutic agent.

Abstract: A transcranial scanning device and method of use is disclosed. The transcranial scanning device is for identifying a neurologic-injury-mechanism and comprises a sensing wand comprising a transducer that produces and receives sound waves. The sensing wand is passed over a head of a patent with cerebral malaria, and sound waves reflect off of blood cells in blood vessels in the head of the patient creating an output sound wave responsive to a blood flow of the patient. The output sound wave is received by the transducer. An output screen coupled to the sensing wand, displays in real time an output waveform corresponding to the output sound wave, wherein the output waveform is indicative of one or more symptoms of cerebral malaria, and wherein said symptoms identify one or more neurologic-injury-mechanism occurring within the patient.

Abstract: Provided are methods for one or more of the following: preventing, inhibiting, disrupting, dispersing or treating a polymicrobial biofilm comprising a Haemophilus bacteria in vitro and/or in vivo, such as in a subject in need thereof. The method comprises or consists essentially of, or yet further consists of contacting the polymicrobial biofilm with: (i) an anti DNA binding and bending protein (DNABII) antibody or a biologically active fragment thereof; and (ii) an anti majority subunit (PilA) of type IV pilus (T4P) antibody or a biologically active fragment thereof. Additionally, the methods may further comprise contacting the polymicrobial biofilm with an antibiotic optionally comprising a ?-lactam antibiotic or a sulfonamide antibiotic. Also provided are compositions and kits suitable for use in the methods.

Abstract: The invention provides for AAV vectors expressing the ANO5 gene and antioxidant therapy as methods of inducing muscle regeneration and a method of treating muscular dystrophy.

Abstract: One aspect of the present disclosure include a method of utilizing an oro-motor device to activate an audio device, the method includes providing an oro-motor device having a sensor and a nipple; producing a signal when the nipple portion present in an infants mouth when the nipple portion yields a first measured pressure over an age appropriate predetermined threshold applied to the nipple portion by the infant; responsive to the signal, playing an age appropriate audio recording for a predetermined duration on an audio device; and raising the age appropriate predetermined threshold to a raised threshold proportionally to a difference between the first measured pressure application to the nipple portion and the age appropriate predetermined threshold.

Abstract: The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.

Abstract: Methods of breaking down a biofilm or inhibiting, preventing or treating a microbial infection that produces a biofilm are disclosed, which involves administration of a polypeptide that has one or more modified HMG-box 1 domains to a subject suffering from the infection or having the biofilm. By competing with microbial proteins that bind to DNA scaffold in the biofilm, these polypeptides destabilize the biofilm leading to destruction and removal of the biofilm by the immune system.

Abstract: Embodiments disclosed herein provide compositions, methods, and uses for recombinant vectors encoding Zika virus (ZIKV) protein subunits, and immunogenic compositions thereof. Certain embodiments provide recombinant vectors encoding ZIKV nonstructural protein 1 (NS 1), and optionally, ZIKV envelope (E) protein and premembrane (prM) protein. Other embodiments provide expression cassettes comprising a promoter operably linked to a polynucleotide that encodes the ZIKV NS 1 protein, and optionally ZIKV E and prM proteins. In some embodiments, the disclosed expression cassettes can be incorporated into a vector to produce a recombinant vector. Also provided are immunogenic compositions comprising one or more recombinant vectors described herein, and methods for inducing an immune response against ZIKV in a subject comprising administering to the subject an immunologically effective dose of an immunogenic composition of the present disclosure.

Abstract: The invention relates to an insertable patient table for a Magnetic Resonance Imaging scanner, wherein the patient table has a length of up to ± 20 % of the length of the bore of the main magnet of the scanner and is adapted for pediatric and/or small patients, and wherein the patient table is adapted to be inserted into the bore of the main magnet without having a stand standing on the floor of an examination room. The invention further relates to a Magnetic Resonance Imaging scanner comprising an insertable patient table and a method for positioning a small and/or pediatric patient within the bore of a main magnet of a Magnetic Resonance Imaging scanner.

Abstract: The invention provides for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: Disclosed are compositions and methods for directing NK cells to the bone marrow through the use of PM21 particles.

Abstract: Methods of and compositions for breaking down a biofilm or inhibiting, preventing or treating a microbial infection that produces a biofilm are disclosed, which involves administration of an interfering agent capable of specifically competing, titrating, or inhibiting the binding of an HU protein to a microbial DNA. By competing with HU proteins that bind to DNA scaffold in the biofilm, these interfering agents destabilize the biofilm leading to destruction and removal of the biofilm by the immune system. Further method and composition aspects are contemplated in relation to infections caused by bacteria that export an HU protein.

Abstract: Certain embodiments of the invention provide EphA4 targeting compounds and compositions comprising a compound described herein. Certain embodiments of the invention also provide methods of treating a neurological disease (e.g., Amyotrophic Lateral Sclerosis, Alzheimer's Disease) or cancer.

Abstract: This disclosure provides isolated or recombinant polypeptides that are useful to vaccinate individuals suffering from chronic/recurrent biofilm disease or as a therapeutic for those with an existing infection. The individual's immune system will then naturally generate antibodies which prevent or clear these bacteria from the host by interfering with the construction and or maintenance of a functional protective biofilm. Alternatively, antibodies to the polypeptides can be administered to treat or prevent infection. Bacteria that are released from the biofilm by our technology are more readily cleared by the remainder of the host's immune system.

Abstract: The present invention relates to IL-8 inhibitor compounds, preferably dual CXCR1/CXCR2 receptor inhibitors, useful in the treatment and/or prevention of some sarcomas, preferably in the treatment and/or prevention of osteosarcoma, Ewing sarcoma, rhabdomyosarcoma or lung metastasis associated thereof.

Abstract: Provided herein are methods for preventing or treating antibiotic induced dysbiosis in a patient by administering to the subject a composition comprising a micro sphere, a bio film-generating probiotic bacterium and a prebiotic, wherein the prebiotic comprises a nutritional supplementation for the probiotic bacterium.