Laboratory of Molecular Genetics

Gene editing techniques have emerged as attractive strategies for correcting diseases with a clear genetic basis. Notably, the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system has emerged as a powerful method for gene inactivation via the formation of small insertions/deletions (indels) and for specific genome editing by dramatically increasing the efficiency of homology-directed repair. The speed and ease of CRISPR/Cas9-mediated gene editing make it an ideal platform for optimizing in vivo disease correction. The ultimate goal of our laboratory is to develop new treatments for various human diseases through genome editing. Finally, we hope to provide the key to combating previously incurable diseases through gene therapy.

Stem cell-based cell therapy is an important field of regenerative medicine with the ultimate goal of enhancing the repair machinery. Our study is based on induced pluripotent stem cells (iPSCs) and direct conversion providing convincing evidence that autologous cell replacement therapies can treat various disorders.To this end, our lab develops innovative cell therapy methods for clinical applications and possible solutions to incurable disease.