New Rochelle, NY, February 15, 2018--China is helping to advance gene and cell therapy and genome editing research and clinical development by creating novel viral and nonviral vectors for gene delivery and innovative applications of CRISPR technology in a broad range of disease areas. An exciting and timely series of original research articles and commentaries documenting the progress, opportunities, and challenges in the Chinese research and biomedical communities is published in a groundbreaking new special issue of Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available free on the Human Gene Therapy website.
The Special Issue on Chinese Gene and Cell Therapy Research is led by Co-Guest Editors Yuquan Wei, MD, PhD, Professor and Director, National Key Laboratory of Biotherapy, West China Hospital, and Vice President of Sichuan University, Chengdu, and De-Pei Liu, MD, PhD, Professor and Former President of Beijing Union Medical College and Chinese Institute of Medical Research, Beijing, and features a comprehensive look at the latest gene therapy research and developments in China.
In the article entitled "Applications of Virus Vector-Mediated Gene Therapy in China," De-Pei Liu and coauthors from Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, highlight the contributions of Chinese researchers in modifying the main groups of viral vectors being used to develop gene therapies. The authors report on progress in China in the application of viral vector-based gene therapy in cancer and monogeneic, neurologic, cardiovascular, liver, and infectious diseases.
Xiawei Wei and coauthors from West China Hospital, Sichuan University and Collaborative Innovation Center for Biotherapy, Chengdu, present "Current Status of Nonviral Vectors for Gene Therapy in China." They describe the progress and challenges associated with efforts to optimize nonviral vectors using structural modifications and opportunities and hurdles related to clinical translation.
In the Clinical Review article "Consideration of Cellular Therapy Products in China," coauthors Chenyan Gao et al. from the China Food and Drug Administration (CFDA), Beijing, discuss the principles on which clinical review of cellular therapy, including CAR-T products in China are based. These include protection of the clinical trial population, pharmacodynamics, pharmacokinetics, dosing, safety, efficacy, and risk management.
Rare diseases research, diagnosis, and related development of new molecular treatments have been lacking in China and held back by inadequate definitions and best practices, orphan drug development, and biobanking to collect samples and genomic data. Shuyang Zhang and colleagues from Peking Union Medical College, National Rare Diseases Registry System of China, Digital China Health Technologies Co., and Rare Diseases Research Center, Chinese Academy of Medical Sciences, Beijing, describe the new opportunities to advance rare diseases medical research and healthcare in China in the article entitled "National Rare Diseases Registry System of China and Related Cohort Studies: Vision and Roadmap."
"This special issue, released coincident with the New Year in China, illustrates the tremendous scientific progress that has been made at certain leading institutions in China working in cell and gene therapy," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "We hope that these particular focused commentaries can provide a roadmap for gene therapy scientists from other parts of the world to identify important achievements and opportunities for future collaboration."