Using pluripotent stem cells to treat sensory and cognitive diseases

More than 275 million people in the world suffer from moderate and severe hearing impairments, most of which are caused by the loss of inner ear hair cells and corresponding auditory neurons. Pluripotent stem cells have the potential for self-renewal and differentiation in vitro, and are divided into embryonic stem cells (ESC) and induced pluripotent stem cells (iPSC). The latest research shows that ESC can be induced into ear progenitor cells and then differentiate into auditory nerve cells. Transplanting ear progenitor cells into deaf gerbils, these cells have the ability to further differentiate and migrate and significantly increase the gerbil's sensitivity to sound.

The article also focuses on another important human perception ability-vision, and highlights the important progress in recent years from PSC differentiation to retinal pigment epithelial cells (RPE). The transplantation of RPE cells differentiated by ESC into the eyes of patients with degenerative vision disease can safely relieve vision degradation. ESC can also differentiate into a three-dimensional optic cup structure containing light-sensitive cells. In addition, multiple studies have established a scheme for PSCs to differentiate into neurons in the cerebral cortex in vitro, and the cerebral cortex is the center of human perception and cognition. These advances provide the basis for the research on the application of pluripotent stem cells to treat human cognitive impairment. It is worth noting that technological breakthroughs in cell trans-differentiation and gene targeting technology will also greatly promote the development of cell therapy. Sensing and recognizing disease-specific iPSCs can also be differentiated in vitro into sensory-related cells that carry disease-causing genes, such as hearing and vision. These "disease-specific" nerve cells can be further used in the study of disease mechanisms. In short, these important milestones of regenerative medicine have marked a further step in the biomedical community's efforts to use stem cells to treat humans' perceived diseases.

Liu Guanghui's research group has been committed to using PSC and gene targeting technology for disease simulation and cell therapy research. He used the iPSC of premature aging in children to study the molecular mechanism of premature aging atherosclerosis (Liu et al. Nature 2011); and the iPSC of Parkinson's patients to study the molecular pathological basis of neurodegenerative diseases (Liu et al. Nature 2012). Researcher Liu Guanghui and Dr. Juan Carlos Izpisua Belmonte of Salk Institute in the United States are the corresponding authors of this article. This work was supported by projects such as the Steering Cell and Regenerative Medicine Strategic Pilot Project of the Chinese Academy of Sciences, the National Youth Thousand Talents Program, and the Fund ’s Excellent Youth Fund.

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