The use of animal models in modeling of human genetic disease has many advantages. In some cases, however, this method may not be applicable due to some limitations, such as differences in tissue composition, anatomy and physiology of humans and animals. Isogenic human disease models are a population of cells that are selected or engineered to model a specific genetic disease, in vitro. They ar...

The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (CRISPR/Cas9) genome editing system has revolutionized the field of for therapeutic applications genetic diseases and cancers, development disease models, identification targets drug development. However, safe efficient delivery CRISPR/Cas9 in vivo remains a significant challenge. Compared to vira...