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: Compositions and methods for producing modified AAV Cap genes and combinatorial libraries of chimeric AAV vectors and virions in an AAV serotype 3 background. Selecting for modified AAV3 virions displaying cell- or tissue-specific tropisms differing from WT AAV3. Using the synthetic combinatorial AAV3 capsid libraries for introducing into a selected target host cells one or more nucleic acid molecules useful in diagnostic and/or therapeutic gene-therapy regimens.

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.

Abstract: A method of carrying out a metathesis reaction includes the combination of at least one alkene or non conjugated diene with a Ruthenium-based catalyst with an cyclic(alkyl)(amino)carbene ligand to form a reaction mixture, heating the reaction mixture to a temperature of 100° C. or greater in the absence of a solvent in bulk conditions, and mechanically stirring the reaction mixture. The reaction can be an ADMET, ROMP, a metathesis ring-closure or an olefin exchange reaction.

Abstract: Aspects of the disclosure relate to compositions and methods for the diagnosis and/or treatment of certain neurodegenerative diseases, for example those diseases associated with repeat-associated non-ATG (RAN) translation proteins, such as Alzheimer's disease (AD). In some embodiments, the disclosure relates to identifying a subject having a RAN protein-associated disease by detecting expression or activity of repeat-associated non-ATG (RAN) translation proteins (e.g., RAN proteins). In some embodiments, the disclosure relates to methods of treating a RAN protein-associated disease by administering to a subject in need thereof an agent that reduces expression or activity of RAN proteins.

Abstract: The present disclosure provides for recombinant AAV therapies for the treatment of vision loss associated with Friedreich's ataxia. These therapies are designed for administration to subjects, such as human subjects, including humans diagnosed with or suffering from Friedreich's ataxia. The disclosed vectors comprise transgenes that encode frataxin protein, such as a FXN trans gene encoding human frataxin protein.

Abstract: Disclosed is a medical instrument that includes a first elongated arm and a second elongated arm pivotally attached at a pivoting joint. The first and second elongated arms include a distal end, a proximal end, an inner surface and an outer surface. The first and second elongated arms define, along respective inner surfaces, opposing gripping portions and opposing cutting edges adjacent to the gripping portions. The first or second elongated arms also include a flange that outwardly deflects from either the first or second distal ends and the first and second elongated arms both include an outwardly deflecting flange. Embodiments enable the cutting of sutures at surgical sites possessing numerous sutures in an expedient manner without the instrument catching the sutures. The instrument also includes functionality to remove staples at a surgical site.

Abstract: The subject invention provides therapeutic compositions, and uses thereof for the treatment or amelioration of symptoms of a disease selected from the group consisting of: Ebola virus infection, HIV infection, ataxia, environmental enteropathy, cancer, hangover, inflammatory disease, and porcine epidemic diarrhea. In preferred embodiments, the composition includes a combination of one or more amino acids selected from the group comprising lysine, aspartic acid, glycine, isoleucine, threonine, tyrosine, valine, tryptophan, asparagine and/or serine.

Abstract: The present application provides kaolin compositions that surprisingly increase the water use efficiency of plants, reduce the occurrence of insects on crop plants, and treat citrus trees infected with HLB.

Abstract: Provided herein are compositions and methods for packaging a recombinant adeno-associated virus (rAAV) particle comprising using inverted terminal repeats (ITRs) and rep genes of different serotypes and/or using chimeric rep genes.

Abstract: Provided herein are AAVrh74 capsid proteins comprising one or more amino acid substitutions or deletions that confer a liver-detargeting property to AAV particles comprising them. Provided herein are AAVrh74 capsid proteins comprising one or more amino acid substitutions that confer an improved transduction efficiency (e.g., in muscle cells). Also provided herein are methods of using AAVrh74 capsid proteins comprising one or more amino acid substitutions or deletions.

Abstract: Aspects of the application relate to methods and compositions for delivering therapeutic nucleic acids to neural cells or tissue in a subject. Additional aspects of the application relate to therapeutic nucleic acids, for example therapeutic ribozymes, that are useful for inhibiting viral reactivation in a subject.

Abstract: The disclosure provides compositions and methods for producing micro-dystrophin proteins, and the use thereof (e.g., in gene therapy). The disclosure also provides skeletal muscle-specific promoters and the use thereof (e.g., in gene therapy). The disclosure further provides catheter-based methods of delivering gene therapy vectors to skeletal muscle and/or cardiac muscle.

Abstract: Methods and compositions useful for delivering a protein to a plurality of nuclei in a cell. Fusion proteins comprising one or more signals useful for promoting delivery of the proteins to a plurality of nuclei in a multinucleate cell. Methods comprising administering isolated nucleic acids to a multinucleate cell, wherein the isolated nucleic acid comprises a sequence encoding a fusion protein, the fusion protein comprising, a protein of interest fused to at least the following migration signals: (a) at least one nuclear export signal (NFS); and (b) at least one nuclear localization signal (NLS) and/or at least one nucleolar localization signal (NoLS). Fusion proteins comprising: (a) a protein of interest; and (b) at least the following migration signals: (i) a nuclear export signal (NFS); (ii) a nuclear localization signal (NLS); and (iii) a nucleolar localization signal (NoLS). Methods of making and using the same. Methods of delivering recombinant genes and/or fusion proteins to multinucleate cells.

Abstract: Disclosed herein are methods and compositions for identifying and/or treating subjects having or likely to have amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). Antibodies specific for one or more di-amino acid repeat-containing proteins are also provided herein.

Abstract: Provided herein are charge complementary peptides that can be optionally coupled to a cargo polypeptide that are capable of self-assembling under stimulating conditions. The charge complementary peptides can be capable of forming supramolecular structures. Also provided herein are methods of using the charge complementary peptides provided herein.

Abstract: The present invention relates to methods of producing compounds of interest in a recombinant microorganism. In particular, the present invention relates to using a recombinant microorganism comprising a heterologous nucleic acid encoding one or more mycosporine-like amino acid (MAA) biosynthetic enzymes (e.g., MysH) to produce compounds of interest. Compositions comprising compounds produced using such methods are also provided herein. The present disclosure also provides methods of preventing sunburn, cancer, and chronic inflammatory diseases by administering such compositions to subjects in need thereof.

Abstract: The disclosure relates to compounds of Formula (I), as well as compositions, methods, uses, and kits comprising the same. The compounds, compositions, methods, uses, and kits provided herein are useful in treating diseases or disorders including cancer, HIV, and angiogenesis-related disorders.