The present inventors report here, in the DFNB9 mouse model (OTOF knock-out mice), the first proof-of-principle that cochlear delivery of a fragmented cDNA via a dual-AAV vector approach can effectively and long-lastingly correct the profound deafness phenotype of these mice when administered well after their auditory system has matured (P30). The present invention therefore concerns a vector system that allows the expression of the full-length Otoferlin polypeptide, or of a functional fragment thereof, in inner hair cells, for use for treating patients suffering from DFNB9 deafness or preventing DFNB9 deafness in patients having DFNB9 mutations, wherein said patients are patients having a developed and mature auditory system, such as new born babies, toddlers, infants, teenagers or adults.

The present disclosure provides compositions with a modulating gene expression and methods for modulating transcription.

An atopic dermatitis model non-human animal, containing a gene mutation in which a complex containing dedicator of cytokinesis 8 (DOCK8) protein, mammalian STE20-like kinase 1 (MST1) protein, and endothelial PAS domain protein 1 (EPAS1) protein is not formed in CD4.sup.+ T cells.

An atopic dermatitis model non-human animal, containing a gene mutation in which a complex containing dedicator of cytokinesis 8 (DOCK8) protein, mammalian STE20-like kinase 1 (MST1) protein, and endothelial PAS domain protein 1 (EPAS1) protein is not formed in CD4.sup.+ T cells.

Disclosed are non-naturally occurring zebrafish, such as transgenic zebrafish, which comprise a mutation in the rhodopsin (rho) gene. Also disclosed are methods of identifying compounds useful in treating retinal-specific defects and disorders, such as degeneration. Further disclosed are methods of identifying mutations in the rhodopsin gene that can cause retinal-specific defects.

Disclosed herein are methods of using probes for high-throughput screening of guide RNA (gRNA) efficiency for Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRiSPR-associated (Cas)-based genome editing systems. Further disclosed herein is a humanized transgenic mouse model that recapitulates the severe DMD pathology of human patients. The mouse model may be used for determining the feasibility of CRISPR-based therapies for the correction of the human dystrophin gene by gene editing and methods of use.

This disclosure relates to a genetically modified rodent and use thereof as a rodent model. More specifically, this disclosure relates to rodent (e.g., mouse or rat) comprising a loss of function mutation in an endogenous Crnn (cornulin) gene, and to use of such a rodent animal as a rodent model of skin inflammation disorders (e.g., psoriasis).

A system is provided comprising a plurality of C. elegans cultures, where each culture comprises a transgenic C. elegans strain that models a mammalian disease or condition. Methods of using a system, e.g., for characterizing microbial strains of a mammalian microbiome and determining whether such microbial strains affect a mammalian disease or disorder.

A method of preparing a ddx27-deletion zebrafish mutant, including: determining a target of ddx27 knockout on a sixth exon of the ddx27 in a zebrafish and designing a gRNA sequence; using primers T7-ddx27-sfd and tracr rev for PCR amplification with a pUC19-gRNA scaffold plasmid as a template; purifying and transcribing the PCR product obtained in vitro to produce gRNA; introducing the gRNA and a Cas9 protein into the zebrafish; and culturing the zebrafish to obtain a zebrafish ddx27 mutant of stable inheritance. In addition, the application also discloses a phenotype of the ddx27-deletion zebrafish mutant, which plays an important role in investigating the biological function.

A system is provided comprising a plurality of C. elegans cultures, where each culture comprises a transgenic C. elegans strain that models a mammalian disease or condition. Methods of using a system, e.g., for characterizing microbial strains of a mammalian microbiome and determining whether such microbial strains affect a mammalian disease or disorder.