Compositions, methods, and kits are provided for assaying calpain-5 activity and inhibition. In particular, novel peptide substrates are provided for detecting calpain-5, measuring calpain-5 activity, and screening for inhibitors of calpain-5 to identify potential therapeutic agents for treating retinal diseases and other diseases associated with calpain-5 hyperactivity. Additionally, novel inhibitors of calpain-5 are also provided.

Provided are a fusion protein that improves gene editing efficiency and an application thereof. The fusion protein comprises a single-stranded DNA binding protein functional domain, nucleoside deaminase and nuclease. According to CBEs, when carrying our base conversion from C-G to T-A, nucleoside deaminase such as cytosine deaminase carries out deamination by using single-stranded DNA as a substrate, and by re-fusing the single-stranded DNA binding protein functional domain on the fusion protein of the nucleoside deaminase and nuclease, the chance of single-stranded DNA being exposed to the nucleoside deaminase is greatly increased, thereby significantly improving base editing efficiency. The present disclosure provides a breakthrough improvement of single-base gene editing technology and can greatly promote the application thereof in aspects such as gene editing, gene therapy, cell therapy, animal model making, and crop genetic breeding.

The invention relates to a genetically modified mouse comprising a heterozygous mutation of Tardbp (TDP-43) gene in that the Asn at amino acid 390 in TDP-43 is substituted with an amino acid that is different from Asn, wherein the genetically modified mouse exhibits Amyotrophic lateral sclerosis (ALS)-like phenotypes, TDP-43 proteinopathies and/or motor neuron degeneration. The invention also so relates to an isolated spinal cord motor neuron differentiated from an embryonic stem cell (ESC) that is obtained from an offspring of a genetically modified mouse according to the invention. Methods for identifying an agent alleviating and/or suppressing ALS-TDP pathogenesis are also disclosed.

Provided is an invention that is based on the novel function of a transcription factor RP58 in cells of the central nervous system. The present invention relates to: a transgenic non-human animal capable of increasing or decreasing the expression of the transcription factor RP58 in cells of the central nervous system of a non-human animal in the nascent stage and/or during and after the developmental stage; and a pharmaceutical composition for use in the treatment or prevention of brain dysfunction, or behavioral disorder, or a disease related thereto, wherein the pharmaceutical composition comprises a transcription factor RP58 protein, or a gene encoding the transcription factor RP58; etc.

Provided are a mouse and a functional activity part thereof, comprising a humanized IL-15 gene; the humanized IL-15 gene comprises a human IL-15 gene segment and a mouse IL-15 gene segment, the human IL-15 gene segment comprises at least a part of exon 4, exon 5, exon 6, exon 7 and exon 8 of the human IL-15 gene, and the mouse IL-15 gene segment comprises exon 1, exon 2 and exon 3 of the mouse IL-15 gene. Also provided are a preparation method and use of the mouse.

The present invention relates to a brain tumor animal model that directly reflects the phenomenon in a human patient and a method of preparing the same, and more specifically, a brain tumor animal model that mutations are introduced into p53, Pten, and EGFR genes, a screening method of a therapeutic agent for a brain tumor using the animal model, and a preparing method thereof.

This disclosure provides a method of treating an infection or inflammation of an eye of a mammal including contacting an eye with a solution comprising sterilized water and protocatechuic acid. The protocatechuic acid may be between about 0.01 wt % and 1.25 wt % in the solution. The solution may include saline. The solution may include ciprofloxacin and/or norfloxacin. This disclosure further provides a method of disinfecting a contact lens including contacting a contact lens with a solution including protocatechuic acid. The solution may include a surfactant. The surfactant may include polyethylene glycol esters of fatty acids, coconut, polysorbate, polyoxyethylene or polyoxypropylene ethers of high alkanes C12-C18. The surfactant may include poly(oxypropylene)-poly(oxyethylene) adducts of ethylene diamine having a molecular weight about 7,500 to about 27,000 wherein at least about 40 weight percent of the adducts is poly(oxyethylene).

Progenitor cells were isolated, purified and expanded using a microfluidic based cell sorting approach. The methods were successfully in purifying cone progenitor cells (CPCs) defined based on a proliferative population expressing cone arrestin and Red/Green (R/G) opsin at greater than 80% using a two multistage approach.

The present disclosure relates to genetically modified non-human animals that express a human or chimeric (e.g., humanized) TNFR2, and methods of use thereof.

The disclosure provides a mouse model of arteriovenous malformation, such as found in Hereditary Hemorrhagic Telangiectasia, that accurately and persistently models the disease progression in various organisms, including humans. The disclosure further provides a mouse comprising a mutant Ephrin pathway gene, such as Alk1, in brain endothelial cells only, and methods of screening for therapeutically useful modulators of Ephrin pathway gene expression or gene product activity useful in treating or ameliorating a symptom of arteriovenous malformation, such as Hereditary Hemorrhagic Telangiectasia or hemorrhagic stroke.