A method for constructing a lung cancer animal model. According to the method, a pathogenic substance of a lung cancer is atomized into atomized particles, and an animal inhales the atomized particles in an inhaling mode, so as to construct a required lung cancer animal model.

Disclosed are methods of administering AAV viral-based vector compositions useful in delivering a variety of nucleic acid segments and compositions comprising an agent, such as an immunosuppressive agent. Methods and compositions comprising a combination therapy are provided. The disclosed AAV vector compositions include those encoding therapeutic polypeptides to selected mammalian host cells for use in therapeutic autoimmune modalities, including, for example, the in vivo induction of immunological tolerance via a liver-directed AAV -based gene therapeutic regimen for treating and/or ameliorating autoimmune disorders such as multiple sclerosis. The compositions comprising an agent may comprise a sphingosine analog, a glucococorticoid, an mTOR inhibitor, and/or a targeted biologic.

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.

A pharmaceutical composition suitable for preventing or treating cardiac arrhythmia is disclosed. The pharmaceutical composition contains, as an active ingredient, a CCN5 protein or a nucleotide encoding the CCN5 protein. The pharmaceutical composition inhibits the pathological activity of CaMKII, which induces cardiac electrical abnormalities that is the main cause of atrial arrhythmia and ventricular arrhythmia, so as to restore the electrical functions, and inhibits the activity of myofibroblasts causing structural abnormalities. Therefore, the pharmaceutical composition can be effectively used in the prevention or treatment of cardiac arrhythmia.

Provided is a method for developing a secondary organ by using a non-human animal in which organ formation is inhibited, for the purpose of establishing a process for producing a functional cell such as a β cell within the body of an animal such as a pig, the method including the step of raising a newborn or a fetus of the non-human animal in which organ formation is inhibited by complementing at least a part of the function of the organ whose formation is inhibited.

Provided herein are screening methods and animal models related to intraocular diseases such as age-related macular degeneration (AMD), for example, for identifying candidate therapeutics for treating or preventing eye diseases, such as AMD. Also provided herein are compounds/compositions that are useful for killing or inhibiting the growth of a microorganism, such as Bacillus megaterium. Further provided herein are methods of using the compounds/compositions for treating infections with a microorganism, such as Bacillus megaterium and for treating or preventing diseases or disorders associated with such infections, such as AMD.

Provided is a method for developing a secondary organ by using a non-human animal in which organ formation is inhibited, for the purpose of establishing a process for producing a functional cell such as a β cell within the body of an animal such as a pig, the method including the step of raising a newborn or a fetus of the non-human animal in which organ formation is inhibited by complementing at least a part of the function of the organ whose formation is inhibited.

Provided is a drug target for vitiligo. The drug target is IFN-γ signaling. It also provides a method for establishing vitiligo animal model, or a vitiligo induction method in an animal, and the established vitiligo animal model. Further, provided is a method for screening candidate drugs for treating vitiligo using the said animal model.

The inventions provide the art with novel treatments of various airway conditions such as asthma wherein pathological production of mucus is implicated. Disclosed are novel inhibitors of miR-200 micro-RNAs, including inhibitors of miR-141. These miRNAs promote pathological mucus production and other airway dysfunction. They may be targeted by antagomirs which disrupt their activity. Additionally, they may be targeted by compositions with disrupt the gene expression of the targeted miR.

Provided are genetically modified non-human animals that express a human or chimeric (e.g., humanized) IL33, and methods of use thereof.