According to a conventional method for treatment of Sandhoff disease and Tay-Sachs disease comprising administering a modified ?-subunit to a patient in the form of a protein, it is necessary that administration be performed frequently. This invention relates to a recombinant adeno-associated virus virion comprising: capsomere comprising a protein capable of forming a virus virion; and a polynucleotide packaged in the capsomere comprising a promoter sequence and nucleotide sequences operably linked to the promoter sequence encoding a first amino acid sequence derived from the amino acid sequence of the ?-subunit of wild-type human ?-hexosaminidase composed of amino acids 55 to 556 in the sequence as shown in SEQ ID NO: 28 by substitution of amino acids 312 to 318 with glycine, serine, glutamic acid, proline, serine, glycine, and threonine in that order and a second amino acid sequence, which is an amino acid sequence of a signal peptide linked to the N terminus of the first amino acid.

The present invention relates to compositions such as cleaning compositions comprising enzymes. The invention further relates to the use of the compositions comprising such enzymes in cleaning processes.

Aspects of the invention relate to recombinant adeno-associated viruses (rAAVs). In some aspects, the rAAVs comprise artificial genetic regulatory elements that modulate transgene expression. In some aspects, the disclosure relates to the treatment of lysosomal storage disorders.

Embodiments of the present disclosure provide a nanoparticle based platform, and nanoallergens for identifying, evaluating and studying allergen mimotopes as multiple copies of a single mimotope or various combinations on the same particle. The nanoparticle is extremely versatile and allows multivalent binding to IgEs specific to a variety of mimotopes, simulating allergen proteins. Nanoparticles can include various molecular ratios of components. For example, the nanoallergens can include about 0.1-40% mimotope-lipid conjugate and about 60-99.9% lipid. The mimotope-lipid conjugate includes a mimotope, a first linker, and lipid molecule. Nanoallergens can be used in in vitro and in vivo applications to identify a specific patient's sensitivity to a set of epitopes and predict a symptomatic clinical response, identify allergen epitopes through blind screening peptide sequences from allergen protein, and in a clinical application similar to a scratch test.

Disclosed herein are recombinant ?-hexosaminidase variant a subunits that form a ?-hexosaminidase variant a subunit homodimer that have optimized properties for use in treating Tay-Sachs disease or Sandhoff Disease.

Embodiments herein include polynucleotides, vectors and methods for the insertion and expression of transgenes. In an embodiment, a polynucleotide is included. The polynucleotide can include a JeT promoter or variant thereof, an intron sequence less than 400 bases in length, and a polynucleotide sequence encoding a polypeptide or protein operatively linked to the promoter. In an embodiment, a recombinant vector is included. The recombinant vector can include a JeT promoter or variant thereof, an intron sequence less than 400 bases in length, and a polynucleotide sequence encoding a polypeptide or protein operatively linked to the promoter. Other embodiments are also included herein.

In alternative embodiments, the invention provides compositions and methods for treating various disorders and conditions in mammals, including chronic disorders in which there is a presence of an abnormal microbiota or an abnormal distribution of microflora in the gastrointestinal tract. In alternative embodiments, the invention provides liquid preparations or formulations derived from a human fecal material (e.g., a stool) processed, e.g., filtered and/or centrifuged, such that all bacteria, fungal spores and viruses are removed, but retaining the native biologically active molecules from the fecal material and bacteriophages. In alternative embodiments, the invention provides a rough-, incomplete- or medium-filtered microbiota which still comprises native physiological components or nutritive agents for the bacteria, e.g., retains native biologically and nutritionally active components. In alternative embodiments, the invention provides a highly filtered or substantially purified microbiota in combination with, or having added back, a liquid preparation or formulation of the invention. In alternative embodiments, the invention provides compositions or formulations where the bacteria, or microbiota, component has been cultured, or cultured under anaerobic conditions, or harvested, stored and/or cultured under anaerobic conditions. In alternative embodiments, the invention provides various additives, compositions and donor restrictions for treating these disorders and conditions.

The present invention concerns genetically modified Mycoplasma bacteria that express gene products having an anti-biofilm activity against a biofilm formed by multiple bacteria such as Pseudomonas aeruginosa and Staphylococcus aureus. Further intended are pharmaceutical compositions comprising genetically modified Mycoplasma bacteria and their use as a medicament.

Methods to introduce highly phosphorylated mannopyranosyl oligosaccharide derivatives containing mannose-6-phosphate (M6P), or other oligosaccharides bearing other terminal hexoses, to carbonyl groups on oxidized glycans of glycoproteins while retaining their biological activity are described. The methods are useful for modifying glycoproteins, including those produced by recombinant protein expression systems, to increase uptake by cell surface receptor-mediated mechanisms, thus improving their therapeutic efficacy in a variety of applications.

Disclosed are novel gene therapy constructs containing both HEXA and HEXB genes to treat GM2 gangliosidoses, including Sandhoff disease and Tay-Sach's disease. Also described are co-treatments using chaperone and anti-inflammatory agents to enhance the effects of gene therapy.