Nonsense-mediated mRNA decay (NMD) polypeptides, nucleic acids encoding NMD polypeptides, and methods of using such polypeptides and nucleic acids in the treatment of ALS and in screening for agents for the treatment of ALS are described.

Methods are provided for treating a subject with a pain condition. Aspects of the methods include administering a gene therapy to the subject and/or a therapeutically effective amount of a composition that includes a gene therapy vector. Aspects of the vectors may include a nucleic acid sequence encoding a K-Cl cotransporter 2 (KCC2) polypeptide, including e.g., full-length and modified versions thereof. Methods are also provided for treating a subject by editing an endogenous KCC2 locus of the subject to encode a modified KCC2 polypeptide. Methods of detecting the presence of a pain condition are also provided, including where a pain condition detected in such methods is treated according to the methods described herein. Also provided are compositions, such as compositions including a gene therapy vector, such as a lentiviral vector, that includes a viral backbone nucleic acid comprising a sequence encoding a full-length human KCC2 polypeptide or a nucleic acid sequence encoding a modified KCC2 polypeptide.

Disclosed are Pear 1 and, serving as a target of fibrotic diseases, applications in a medicament for treating human Pear 1-related diseases. Also disclosed are a Pear 1-targeting antibody or a mutant of same or a composition comprising same and applications thereof. Disclosed are a humanized Pear 1 transgenic mice model, a construction method for same, and applications thereof. The technical solution of the present invention regulates fibroblast activation and has broad application prospects in treating fibrotic diseases and accompanying debilitating diseases.

An object of the present invention is to produce a mammalian organ having a complicated cellular composition composed of multiple kinds of cells, such as kidney, pancreas, thymus and hair, in the living body of a non-human animal. The inventors of the present invention applied the chimeric animal assay described above, to a novel solid organ production method. More specifically, the inventors has shown that a model mouse which is deficient of kidney, pancreas, thymus or hair due to the dysfunction of the metanephric mesenchyme that is differentiated into most of an adult kidney, is rescued by blastocyst complementation by the chimeric animal assay, and whereby a kidney, a pancreas, thymus or hair can be newly produced.

Provided is use of thiamine pyrophosphokinase TPK as a target in the treatment of Alzheimer's disease; and AD symptoms due to the inhibited TPK can be prevented by promoting the kinase activity and/or expression level of TPK protein in brain with TPK as a target.

Provided is a polynucleotide in which an N-terminal region of TIF1y gene is codon-optimized, a recombinant vector including the polynucleotide, and a use thereof.

Provided here are certain recombinant HIV compositions and animal models to evaluate prophylactic and therapeutic antiviral compositions.

Provided herein are methods for creating an animal model for the study of dry eye and methods of using such animal models to test candidate treatments for ocular conditions and determine the efficacy of such treatments.

The present invention relates to the field of therapeutic use of proteins, genes and cells. More specifically the invention relates to therapy based on the biological function of a secreted therapeutic protein, NsG33, in particular for the treatment of disorders of the nervous system. NsG33 is a nerve survival and growth factor with antiapoptotic effects on a cell line with neuronal potential and with neuroprotective and/or neurogenesis effects on a neural precursor cell line and on primary striatal cultures. The invention also relates to novel bioactive NsG33 polypeptide fragments and the corresponding encoding DNA sequences.

The present invention includes compositions and methods for treating diseases or disorders associated with pathological calcification or pathological ossification. In certain embodiments, the diseases or disorders are selected from the group consisting of Generalized Arterial Calcification of Infancy (GACI), Idiopathic Infantile Arterial Calcification (IIAC), Ossification of the Posterior Longitudinal Ligament (OPLL), hypophosphatemic rickets, osteoarthritis, calcification of atherosclerotic plaques, PXE, hereditary and non-hereditary forms of osteoarthritis, ankylosing spondylitis, hardening of the arteries occurring with aging, calciphylaxis resulting from end stage renal disease and progeria.