The present application discloses methods of correcting a gene defect in a cell, methods of treating a patient having a disease or disorder characterized by a gene defect, methods of preparing a chimeric antigen receptor T cell, as well as systems for correcting a gene defect in a cell, ex vivo modified cells, and related compositions.

The present invention relates to a method for producing an animal model of preterm birth and an animal model of preterm birth produced by the method. The animal model of the present invention can be effectively applied to investigate the causes and symptoms of preterm birth induced by cervical injury. The mortality rate of the animal model according to the present invention is low until preterm birth despite its induced preterm birth. In addition, the animal model of the present invention is produced in a higher yield than any other existing model. Furthermore, the preterm birth of the animal model according to the present invention is induced at a desired time point. Due to these advantages, the animal model of the present invention can be effectively applied to investigate the causes and mechanisms of preterm birth. The mortality rate of premature neonates born from the animal model of the present invention is considerably low and the premature neonates are immature. Therefore, the animal model of the present invention can be effectively applied to studies on complications of premature neonates.

The invention provides compositions and methods useful for the treatment and prevention of conditions associated with short telomere length.

Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.

Described herein are transgenic mice for testing immunogenicity and protective efficacy of a wide variety of therapeutic agents and vaccines, determining allograft rejection, and developing monoclonal antibodies and generating hybridomas. Methods of generating a transgenic mouse is also described. Described herein are mouse models capable of expressing B cell, a T cell, a monocyte, a macrophage, a dendritic cell, a NK cell, a iNKT cell, an innate lymphoid cell, a microglia cell, a red blood cell, which can develop into a functional human immune system.

The invention provides novel engineered CRISPR/Cas effector enzymes, such as Cas13 (e.g., Cas13d, Cas13e, or Cas13f) that substantially maintain guide-sequence-specific endonuclease activity and substantially lack guide-sequence-independent collateral endonuclease activity compared to the corresponding wild-type Cas. Also provided are polynucleotides encoding the same, vectors or host cells comprising the polynucleotides or engineered Cas, and method of use, such as in RNA-based target gene transcript knock down.

Transgenic non-human animal models for cellular senescence are provided herein, as are methods and materials for making and using the transgenic non-human animal models. For example, a p21-Cre mouse model for cellular senescence is provided herein.

Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.

An auxin-inducible degron system kit that controls degradation of a target protein in a non-plant-derived eukaryotic cell, the kit containing a first nucleic acid that encodes a mutant TIR1 family protein having a mutation at an auxin-binding site, an auxin analog that has an affinity to the mutant TIR1 family protein and a second nucleic acid that encodes a degradation tag containing at least a part of an Aux/IAA family protein and having an affinity to a complex of the mutant TIR1 family protein and the auxin analog.

Disclosed is a method for culturing urine-derived kidney stem cells, which belongs to the field of cell biology. The method comprises the following steps: isolating cells from the urine, and then culturing the cells with a culture medium of urine-derived kidney stem cells on feeder cells to obtain the urine-derived kidney stem cells, wherein the feeder cells are fibroblasts, and the culture medium of urine-derived kidney stem cells contains 200-300 mL of DMEM medium, 200-300 mL of F12 medium, 20-70 mL of fetal bovine serum, 0.2-2 mM of L-glutamine, 1-14 ng/mL of insulin, 0.1-1 ng/mL of epidermal growth factor, 5-30 μg/mL of adenine, and 2-20 μg/mL of hydrocortisone. By using the method, kidney stem cells with high proliferation capacity and specificity can be obtained and applied, and thus the regenerative outcome of the kidney tissue after injury can be improved.