iPSC-Derived Cell Models of Human Single-Gene Disorders

TOPICAL COLLECTIONS

Guest Editors

Lidan Hu

Zhejiang University School of Medicine, the Children’s Hospital

Dr. Lidan Hu earned her PhD in “protein structure and function, RNA decay, and cell bodies” from Tsinghua university in 2017 (China). During her post-doctorate at Tsinghua University, she discovered various compounds which could regulate the dynamic of cell bodies, such as aggresome, stress granule and processing bodies. In 2020, Dr. Hu joined National Clinical Research Center for Child Health (Zhejiang university) and investigated the pathomechanism of genetic diseases. Her research focuses on the mechanism of monogenetic diseases, their cure, and mRNA metabolism (decay and RNA granules).

Xiaoling Guo

Wenzhou Medical University

Dr. Guo is a visiting scholar at the Harvard Stem Cell Institute. He completed his PhD at Jinan University in 2016 under Dr. William T. Pun. Prior to this he completed his MS in Genetics and worked on human pluripotent stem cells under the guidance of Prof. Jian-Su Chen.

Collection Overview

Induced pluripotent stem cells are produced by reprogramming terminally differentiated somatic cells with specific transcription factors. Induced pluripotent stem cells have a strong ability to differentiate and regenerate. It can differentiate into various cell types required by the various organs and tissues of the human body. It is similar to embryonic stem cells in terms of their pluripotency and differentiation potential, pluripotent gene expression, epigenetic embryoid, and teratoma formation.

Unlike embryonic stem cells, induced pluripotent stem cells are derived from genetic editing, so there are no social, ethical, or moral constraints in terms of their origin because they come from adult individuals. It is found to reduce immune rejection in clinical applications. In addition, induced pluripotent stem cells have low immunogenicity and are widely used in regenerative medicine, screening, and developing new drugs.

The purpose of this Methods Collection is to collect original research, generate homologous control iPS cell lines through reprogramming or CRISPR / Cas9 or Bemax gene editing system, and induce these iPS cells to differentiate into desired target cells or organs to establish cell models and apply them to organ regeneration research and drug development.