The present disclosure provides a pharmaceutical composition for treating Alzheimer's disease and use thereof, the pharmaceutical composition containing, as an active ingredient, stem cells (SC) having high adaptability under hypoxic conditions. Particularly, the present disclosure discloses a pharmaceutical composition for alleviating or treating the symptoms of Alzheimer's disease, the pharmaceutical composition containing, as an active ingredient, mesenchymal stem cells (MSC) which have high adaptability or viability in hypoxic environments and comprise one or more knock-out HIF1AN genes.

The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

Among the various aspects of the present disclosure relates to methods and compositions useful in convert inflammatory dietary fatty acids (omega 6) to anti-inflammatory fatty acids (omega 3). Specifically, the present disclosure provides methods and compositions for increasing n-3 desaturase expression or activity in a subject. The disclosure provides various compositions for providing increased n-3 desaturase expression or activity including a variety of viral vector and engineered microorganisms The disclosure provides, in part, methods for reducing local and/or systemic inflammation, improving wound healing, preventing premature cellular enescence, treating or preventing a disease, disorder, or condition related to inflammation or obesity and treating or preventing arthritis.

The invention relates to animal models, and in particular to novel in vivo animal models for neurodegenerative disorders, such as Alzheimer's disease, Parkinson's disease or Motor Neurone Disease. The invention extends to methods for providing such models. The invention also provides animal models per se and methods for investigating the underlying mechanisms occurring in such neurodegenerative disorders, in particular, Alzheimer's disease, and also extends to models, methods and assays for testing pharmacological test compounds, which may modulate neurological processes, and for drug screening for use in treating neurodegenerative diseases.

The disclosure relates to a sarcoidosis animal model and methods of inducing sarcoidosis in an animal. The disclosure also relates to methods of producing an in vitro granuloma, and methods of using the sarcoidosis animal model. Disclosed herein are animals comprising one or more granulomas, wherein the one or more granulomas comprise Mycobacterium abscessus cell wall microparticles.

Provided are a modified-type neuraminidase, a gene encoding the modified-type neuraminidase, a combination of the modified-type neuraminidase and cathepsin A, a combination of the gene encoding the modified-type neuraminidase and a gene encoding cathepsin A, a vector including said genes, and a pharmaceutical composition containing same. The pharmaceutical composition can be used for the therapy of lysosomal storage disease.

Polynucleotide constructs and multifunctional engineered natural killer (NK) cells expressing such constructs are provided for the treatment of cancer and, in particular, glioblastoma. The constructs are a fusion of a first binding domain that targets at least one cognate ligand on a target cell, a second binding domain specific for an adenosine producing cell surface protein of the target cell or an adenosine-intermediary producing cell surface protein of the target cell and a cleavable linker, and a third binding domain specific for a cancer-associated antigen. Pharmaceutical compositions of the engineered NK cells are also provided, as well as methods of treating glioblastoma using such pharmaceutical compositions alone and in addition to autophagy inhibitors.

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.

A preparation method of an anti-PD-1/PD-L1 monoclonal antibody (mAb)-induced autoimmune myocarditis model is provided, including: mediating a model with adeno-associated virus 9 (AAV9) to achieve the high expression of PDL1 in a myocardial tissue, and applying an anti-PD-1/PD-L1 mAb to the model with high PDL1 expression in the myocardial tissue for modeling. The present disclosure also provides use of an animal model prepared by the preparation method. The model prepared by the present disclosure truly simulates the pathogenesis and clinical course of autoimmune myocarditis in a patient administered with an anti-PD1/PD-L1 mAb, is close to a pathophysiological status of a clinical patient, has a high modeling rate, and can be dynamically monitored.

Disclosed are methods for making a vascularized hollow organ derived from human intestinal organoid (HIOs). The HIOs may be obtained from human embryonic stem cells (ESC's) and/or induced pluripotent stem cells (iPSCs), such that the HIO forms mature intestinal tissue. Also disclosed are methods for making a human intestinal tissue containing a functional enteric nervous system (ENS).