The present invention relates to a sorLA-based drug screening platform for use in screening compound libraries for an effect on endosomal activity.

Disclosed herein are methods for treating a subject with an alkaline phosphatase deficiency, further comprising monitoring additional analytes, e.g., calcium, parathyroid hormone and/or vitamin D, with treatment modifications as indicated by the levels, e.g., serum levels, of the additional analytes.

The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.

The invention provides a method for inducing gelation and biomimetic mineralization of a silk fibroin solution by alkaline phosphatase. A micromolecular polypeptide that is sensitive to ALP and has good biocompatibility and self-assembly property is introduced as a gelator precursor, which can remove a phosphate group under the catalytic action of ALP to generate NY, to trigger supramolecular self-assembly, and therefore SF co-self-assembly is synergistically induced, finally resulting in rapid formation of SF hydrogel. ALP wrapped in an SF-NY hydrogel network still retains its catalytic activity and catalyzes beta-glycerophosphate to release free phosphate ions, so that formation of apatite minerals is induced in the gel. The biomimetic mineralized SF gel can be used as a biomimetic scaffold to promote the adhesion, proliferation and osteogenic differentiation of rat bone marrow mesenchymal stem cells in vitro, and can also promote the natural healing of femoral defects in a rat model.

Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.

The present disclosure provides a technique for quantitatively detecting alkaline phosphatase (ALP) activities in seawater and other aquatic environments, based on surface-enhanced Raman spectroscopy by taking 5-bromo-4-chloro-3-indolyl phosphate (BCIP) as a substrate and dimethyl sulfoxide (DMSO) as an internal standard. Results show that ALP activity has a good linear correlation with the intensity ratio of a characteristic Raman peak to that of the internal standard peak (600 cm.sup.−1/677 cm.sup.−1) (R.sup.2=0.977). The technique was successfully applied to detect ALP activity of a seawater sample. By extension this technique can also be used in detecting the activity of other microbial extracellular enzymes (e.g., aminopeptidase) in seawater and thus, lays a solid scientific foundation for in-situ detection of the activities of other extracellular enzymes in seawater and other aquatic environments.

The present invention provides a cell-based assay for measuring T cell activation mediated by a T cell-dependent bispecific antibody (TDB). In some aspects, the assay is useful for detecting a TDB in a composition, quantitating the amount of TDB in a composition, determining the potency and/or specificity of a TDB, or determining if a population of cells expresses a target antigen. Compositions and kits are also contemplated.

The disclosure provides a method of producing recombinant alkaline phosphatase comprising: (i) inoculating Chinese Hamster Ovary (CHO) cells expressing recombinant alkaline phosphatase in culture medium; (ii) culturing the CHO cells in the culture medium at a temperature of about 37° C.; (iii) adding a combination of nutrient supplements to the cell culture of (ii) at least one day after inoculation, the combination comprising (a) a first animal-derived component-free (ADCF) nutrient supplement comprising one or more amino acids, vitamins, salts, trace elements, poloxamer and glucose, wherein the first ADCF nutrient supplement does not comprise hypoxanthine, thymidine, insulin, L-glutamine, growth factors, peptides, proteins, hydrolysates, phenol red and 2-mercaptoethanol; and (b) a second ADCF nutrient supplement comprising one or more amino acids, wherein the second ADCF nutrient supplement lacks hypoxanthine, thymidine, insulin, L-glutamine, growth factors, peptides, proteins, hydrolysates, phenol red, 2-mercaptoethanol and poloxamer; (iv) decreasing the temperature of the cell culture of (iii) to about 30° C. about 80 hours to 120 hours after the inoculation; and (v) isolating the recombinant alkaline phosphatase from the cell culture of (iv) by at least one chromatography step.

Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.

The present invention is directed to bispecific humanized PLAP (placental alkaline phosphatase)-CD3 epsilon chain (CD3e) antibodies. The present invention is further directed to a method for treating PLAP-positive cancer cells by administering the bispecific PLAP-CD3e antibody to the patients.