Abstract
Abstract:In the 1960 of the 20th century, scientists firstly advanced
the concept of using gene therapy to cure the human genetic disease,
which aimed to carry out precisely site-specific modification on the
genome for obtain long-term therapeutic effect in patients. In the next
50 years, scientists have developed many new gene editing technologies
including zinc-finger nucleases (ZFNs), transcription activator-like
effector nucleases (TALENs), and clustered regularly interspaced short
palindromic repeats (CRISPR), etc. These new technologies have brought
milestone breakthroughs in gene therapy era. In this review, we focus on
how flexible the CRISPR system used in human genetic disease-focused
research, gene therapy strategies, clinical trials and discuss some of
the major challenges and significant benefit for its future use in
patients.