Provided is a pharmaceutical composition for treating a degenerative brain disease, including a glycine transporter as an active ingredient. A composition including, as an active ingredient, a glycine transporter protein, a fragment thereof or a nucleic acid molecule encoding the protein or the fragment thereof, a vector including the nucleic acid molecule, or a cell transformed with the vector including the nucleic acid molecule, according to an embodiment, not only can achieve excellent effect(s) of inhibiting amyloid-beta aggregation and/or degrading aggregated amyloid-beta, but also degrades tau protein (and/or inhibition of the aggregation thereof), inhibits the hyperphosphorylation of tau protein, and has excellent blood-brain barrier permeability, thus making it possible to successively act on brain tissues. Therefore, the composition can be effectively applied to the prevention and/or treatment of various degenerative brain diseases associated with amyloid-beta aggregation, tau protein aggregation, and/or hyperphosphorylated tau protein.

Nucleic acids encoding modified myosin heavy-chain-associated RNA transcripts are provided. The modified myosin heavy-chain-associated RNA transcripts belongs to a cluster of long noncoding RNAs (lncRNA) and bind to chromatin remodeler Brg1 to inhibit Brg1's genomic targeting and gene regulation function. The modified myosin heavy-chain-associated RNA transcripts expressed in an individual inhibit Brg1's gene regulation function and protect the heart of the individual from myopathy and failure. One of the modified heavy-chain-associated RNAs is a 400 base pair fragment segmented from a natural 779 base pair sequence of Mhrt (Mhrt779) and has the same cardioprotective effects as the Mhrt779.

The present invention pertains to a silkworm-type biotinylated CTLD14 or sRAGE and a method for manufacturing the same. One embodiment of the present invention provides a method for manufacturing biotinylated proteins, wherein the method includes A) a step for inserting a nucleic acid molecule for coding biotin ligase and protein in a coexpressable manner into a silkworm or a living organism that imparts sugar chains that are the same as the sugar chains of the silkworm, B) a step for causing the biotin ligase and protein to be expressed by disposing the silkworm or the living organism that imparts sugar chains that are the same as the sugar chains of the silkworm to conditions with which the nucleic acid molecule will carry out expression, and C) a step for administering biotin to the living organism and obtaining the biotinylated protein.

The present invention relates to an animal model for oxidative stress research and use thereof, and more specifically, the present invention can utilize a mutant of RCAT having a regulatory function for an antioxidant stress regulator in Caenorhabditis elegans and a human cell line expressing RCAT as animal and human cell line models for oxidative stress research, using the mutant and the human cell line.

Among the various aspects of the present disclosure is the provision of engineered cells, animal models, and uses thereof for modeling low grade glioma (LGG). An aspect of the present disclosure provides for a population of cells engineered to silence, downregulate, knock out, or reduce or knock down Cxcl10 expression. Another aspect of the present disclosure provides for an animal engineered to be deficient in Cxcl10, downregulate or reduce expression of Cxcl10, knock out Cxcl10, or knock down Cxcl10 (e.g., Cxcl10.sup.−/− mice). Yet another aspect of the present disclosure provides for a method of growing tumor cell lines or patient-derived xenografts for LGG tumors in an animal (e.g., mouse, rat) including providing a mouse or rat harboring somatic homozygous deletion in the Rag1 or Cxcl10 gene, and implanting an amount of the cells in mice sufficient to grow a tumor.

The present invention relates to a HSP27 mutation (S135F) mediated Charcot-Marie-Tooth disease (CMT) animal model. Particularly, the vector expressing mutant HSP27 protein wherein the 135.sup.th serine is substituted with phenylalanine has been injected in the mouse zygote and then the mouse harboring the expression vector was selected. The selected mouse was confirmed to display Charcot-Marie-Tooth disease phenotype, so that the animal model was expected to be efficiently used for the evaluation of the effect of Charcot-Marie-Tooth disease treating material candidates.

Provided herein are methods and compositions related to non-human animals that express exogenous Terminal Deoxynucleotidyltransferase (TdT).

CRISPR/Cas9 is becoming an increasingly important tool to functionally annotate genomes. However, since genome-wide CRISPR/Cas9 libraries are mostly constructed in lentiviral vectors, in vivo applications are severely limited due to difficulties in delivery. Here we examined the piggyBac (PB) transposon as an alternative vehicle to deliver a guide RNA (gRNA) library for in vivo screening. Although tumor induction has previously been achieved in mice by targeting cancer genes with the CRISPR/Cas9 system, in vivo genome-scale screening has not been reported. With our PB-CRISPR libraries, we conducted an in vivo genome-wide screen in mice and identified genes mediating liver tumorigenesis, including known and novel tumor suppressor genes (TSGs), Our results demonstrate that PB can be a simple and non-viral choice for efficient in vivo delivery of CRISPR libraries.

Hoxb5 identifies long-term hematopoietic stem cells. Expression of Hoxb5 distinguishes between LT-HSCs and non-LT-HSCs, and the marker identifies substantially all LT-HSC in the bone marrow. By utilizing fluorescent proteins under the endogenous expression control of Hoxb5, LT-HSC can be monitored and isolated, including without limitation detection and monitoring of HSC in bone morrow; production of LT-HSC from pluripotent stem cells such as iPS cells; for analysis of early stage LT-HSC; in screening methods for expansion and manipulation of LT-HSC, and the like.

Methods of generating functional human organs and tissue in animal bodies suitable for transplantation into human subjects are provided. In particular, the contribution of human donor cells to tissues and organs can be increased in interspecies host embryos by knocking out a growth factor receptor gene such as the insulin-like growth factor 1 receptor or insulin receptor gene. Almost entirely donor-derived functional organs and tissue can be generated by using this method. The methods described herein are useful for generating human organs and tissue in animals and may be helpful for overcoming the current problems with organ shortage for transplantation therapy. Additionally, such organs and tissue can be used in drug discovery, drug screening, and toxicology testing.