Abstract
Rapid advancements in gene therapy have greatly facilitated the treatments and cures for genetic disorders. In China, there are approximately 200 000 newborn babies each year with genetic disorders, 22% of which are caused by monogenic mutations. Birth defects, endocrine or metabolic diseases, nervous system diseases, and blood system diseases are the four most common genetic disorders. Additionally, gene therapies such as chimeric antigen receptor (CAR) T-cell immunotherapy have been increasingly reported as effective treatments for diseases such as cancer and HIV infection.
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