Abstract: The present invention provides novel methods and kits for determining whether a subject is suffering from cisplatin-containing resistant ovarian and methods for treating and reversing resistance.

Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.

Abstract: The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: A sanitizable audio device for use in neonatal care and method of use are provided herein. The audio device comprises a smooth shell defining an outer surface and an inner surface. The outer surface forms a curved continuous smooth surface. The inner surface supports electronic elements. The shell defines one or more switch apertures, housing one or more actuatable electrical switches, in communication with the electronic elements. The method of use comprises generating an audio output including selecting content that is age appropriate for a particular infant, altering a sound emission of a recording of a care-giver of the particular infant, and programming a music player to emit the audio output below an age dependent volume, for an age dependent number of intervals per day and per week, for an age dependent duration.

Abstract: An adjustable protective mouth assembly and method for protecting the surroundings adjacent to the oral cavity of a patient is provided. The adjustable protective mouth assembly includes a channeled body comprising first and second legs spaced and integrally coupled by a connecting portion. The channeled body is for inserting into the oral cavity of a patient during use. The channeled body further comprises first and second ends, such that the channeled body of the first end is constructed to be received and nest within the channeled body of the second end, forming a connection therebetween.

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 cannot form functional biofilms are more readily cleared by the remainder of the host's immune system and/or traditional antibiotics.

Abstract: This disclosure provides pharmaceutical compositions and purified or isolated naturally occurring exosome products that have therapeutic use for treating an unmet medical need. The exosome compositions contain an effective amount of exosomes isolated from a body fluid of a non-diseased subject. The compositions are useful in the treatment of a variety of fibrotic diseases.

Abstract: The current disclosure relates to methods, compositions and kits for detecting modified adenosine in a target RNA molecule. Aspects relate to a method for detecting modified adenosine in a target ribonucleic acid (RNA) comprising contacting the target RNA with an adenosine deaminase enzyme (adenosine deaminase, RNA-specific) to generate a target RNA with deaminated adenosines and sequencing the target RNA with deaminated adenosines; wherein the modified adenosine is detected when the nucleotide sequence includes adenosine within a m6A motif.

Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.

Abstract: This disclosure provides methods for determining whether a subject is suffering from a liver cancer, such as hepatic carcinoma, comprising determining the expression level of miRNA-33b and/or miRNA46-a, in a sample of exosomes enriched from a body fluid of the subject, wherein low expression of the miRNA-33b and/or miRNA-46a as compared to a control is indicative of the subject suffering from a liver cancer, e.g., hepatic carcinoma. The subject may also be suffering from a fibrotic disease, liver fibrosis, and/or is or was infected with hepatitis B.

Abstract: The present disclosure provides a novel method for monitoring the conversion of oral precancerous lesions to oral cancers, including monitoring changes in oral exosome concentrations in a subject, thereby determining the state of the disease and the risk of carcinogenesis. The disclosure also relates to a detection kit for use in the above method.

Abstract: The present invention relates to Adeno-associated virus 9 methods and materials useful for systemically delivering polynucleotides across the blood brain barrier. Accordingly, the present invention also relates to methods and materials useful for systemically delivering polynucleotides to the central and peripheral nervous systems. The present invention also relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as spinal muscle atrophy and amyotrophic lateral sclerosis as well as Pompe disease and lysosomal storage disorders. Use of the methods and materials is also indicated, for example, for treatment of Rett syndrome.

Abstract: Disclosed are approaches to determining a sensitivity of a bacterium to a given antibiotic and generating targeted treatments based on the sensitivity. One or more antibiotics may be selected for a chronic/recurrent infection resulting from a biofilm so as to reduce dose and or length of course of antibiotic treatment. For example, if a bacterial pathogen is determined to be sensitive to an antibiotic in its planktonic form but resistant to that antibiotic in its biofilm form, then the biofilm may be dispersed or disrupted from the biofilm residence in order to clear the infection. In various embodiments, a dispersal or disruption method and/or agent may be determined based at least in part on a rate of bacterial release from a biofilm that sensitizes the pathogen to a chosen antibiotic.

Abstract: The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nuclear RNAs to induce DUX4 exon-skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.

Abstract: This disclosure provides kits and methods for determining whether a subject is suffering from a liver disease, and/or whether the subject will be a responsive to a therapy for a liver disease. The disclosed methods comprise determining the miR profile of exosomes isolated from a body fluid of the subject.

Abstract: Provided herein are derivatives of HMGB1 that have been engineered to possess the same efficacious anti-bio film activity but are smaller and do not induce inflammation.

Abstract: Provided herein are methods to inhibit or disrupt a bio film comprising contacting the bio film with an agent that cleaves the Holliday junction (HJ) structure in the bio film.

Abstract: Provided herein are recombinant AAV vectors encoding a GALT protein. Also provided in this disclosure are compositions and methods for the treatment of galactosemia.

Abstract: Provided herein are prenatal methods comprising administering an effective amount of a biocompatible microsphere comprising a biofilm-generating probiotic bacterium, a prebiotic, and/or a prebiofilmic.

Abstract: Provided herein are methods and compositions for disrupting biofilms in vitro and in vivo. Also disclosed are antibodies comprising a specified heavy chain (HC) immunoglobulin variable domain sequence and/or a specified light chain (LC) immunoglobulin variable domain sequence.