Establishment of patient-derived iPSC Patient-derived induced pluripotent stem cells (iPSC) refers to the reprogramming of disease-specific iPSCs in vitro using patient somatic cells, and then differentiation of these iPSC into disease-related functional cells, such as neuron, etc. The disease-specific genotype and phenotype were reproduced in these differentiated cells, which can be studied on the pathogenesis to provide new ideas for developing new therapeutic drugs.
The application of patient-derived iPSC Due to the scarcity of human cells and the differences between individuals, the development of drugs around the world mainly rely on animal experiments, cancer cell lines, and a limited number of dead donated cells. These models directly lead to the high failure rate of new drug development in the clinical trial phase. The emergence of iPSC technology makes the industrial production of various human cells possible. The establishment of an iPSC cell bank containing a variety of disease types, and then differentiation into functional target cell types will greatly improve the efficiency of new drug development and greatly reduce the R & D cost.
Since 2008, there have been more and more studies on disease-specific iPSC, involving genetic diseases of neuron, blood, heart, pancreatic islets, liver, and many other human tissues and organs. The types of diseases include amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), Parkinson's disease (PD), Huntington's disease (HD), schizophrenia, premature aging syndrome, Fanconi anemia, Familial dysautonomia, type I diabetes, dyskeratosis congenita, metabolic liver diseases, etc.
Scope of application (A) New drug development.
(B) Drug safety assessment.
(C) Study on the disease pathogenesis.
(D) Gene therapy.
