Described herein are methods and compositions for autocatalytic genome editing and neutralizing autocatalytic genome editing. The autocatalytic genome editing may be based on genomic integration of a construct containing multiple elements or on a trans-complementation approach, in which genetic elements can be propagated separately. The disclosure provides a method for autocatalytic genome editing based on the CRISPR/CAS9 system, and methods of use thereof, in animals, humans, and plants for eliminating pathogens, targeting suppression of crop pests, strategies to combat virus (e.g., HIV) and other diseases (e.g., cancer) caused by retrovirus, as well as to generate homozygous mutations that are transmitted to nearly all offspring.

Two or more conditional, dominant, lethal gene expression systems provide high levels of penetrance in insects. Lethality is induced at an earlier stage of development and the risk of biochemical resistance is reduced, as compared to a single insect conditional, dominant, lethal gene expression system. The invention is useful for the control of insect populations.

Embodiments of the disclosure include methods and compositions related to the proliferation of cardiomyocytes. In particular embodiments, the proliferation of cardiomyocytes is facilitated upon exposure of the cardiomyocytes to agents that affect the sequestering of Yap by dystrophin glycoprotein complex, such agents including those that inhibit DAG1, Yap, or the interaction thereof.

The present invention relates to a factor VIII or factor IX gene knockout rabbit, a method for preparing the same and a use thereof and, more particularly, to a transgenic rabbit whose factor VIII or factor IX gene has been knocked out through the CRISPR/Cas9 system, a method for preparing the same and a use thereof. According to the present invention, in the transgenic rabbit, whose factor VIII and/or factor IX gene has been knocked out, the functions of factor VIII and/or factor IX, which are proteins that perform critical functions for the development of hemophilia, are inhibited, such that the transgenic rabbit is useful for the development of hemophilia treatments.

A method for optogenetics experiments, based on wavefront shaping and including: calculating the transmission matrix between an input end and an output end of the multimode fiber under a fixed shape; implanting the output end into an intracranial space of an experimental subject; and performing wavefront compensation to a light to be input into the input end, according to the spatial position of the optical stimulation and the transmission matrix of the multimode fiber, to form a compensated expanded light, and inputting the compensated expanded light from the input end into the multimode fiber, such that the compensated expanded light, after being transmitted by the multimode fiber to the output end and output from the output end, is capable of focusing at the spatial position of the optical stimulation.

The present invention relates to a method for treating or alleviating an osteoporosis in a subject. The method comprises steps of identifying the subject having the osteoporosis, and administering to the subject an effective amount of a composition that increases a level of Discoidin Domain Receptor 1 (DDR1) protein in the subject.

Inhibitors of miRNA-128 capable of promoting cardiomyocyte mitotic cell proliferation and methods effective for regeneration of heart tissue.

A liquid suspension product comprising a recombinant adeno-associated virus (rAAV) having an AAV8 capsid which is suitable for intra-retinal injection is provided herein. Also provided herein are liquid suspensions containing these rAAV8.aVEGF and methods of using same for treatment of wet AMD and other ocular conditions.

The present invention relates to the field of fibrosis and inflammation and more particularly to the use of ADAM12 (A Disintegrin and Metalloproteinase 12) inhibitors to prevent or treat inflammation-induced fibrosis. The present invention also relates to the use of ADAM12 as a marker for inflammation-induced fibrosis and to the ablation of ADAM12 expressing cells as therapeutic approach to interfere with the development of pro-fibrotic cells.

Disclosed herein, are inducible immunodeficient animals and methods to make them by adding an IL2Rg/RAG2 rescue cassette (RG-reg) or an IL2Rg/RAG2/FAH rescue cassette (FRG-reg) to a line of IL2Rg/RAG2 knockout (RG-KO) or IL2Rg/RAG2/FAH knockout (FRG-KO) swine. The rescue cassette enables line breeding of immunocompetent (regRG-KO) or (regFRG-KO) swine for rapid propagation. The rescue cassette can be excised, specifically in germ cells of regRG-KO or regFRG-KO swine, such that offspring of animals do not possess the rescue cassette and are immunodeficient. The immunodeficient swine also provide host embryos having genetic ablations to provide a niche for organ complementation by human stem cells.