Genome editing technologies and their potential to treat neurologic disease.

摘要: Genome editing refers to a process of making precise and permanent changes in the genetic code cells, tissues, whole organisms. The first step this is program an enzyme called nuclease bind DNA sequence, whereby will cut DNA. This double-stranded break turn induce cell make repair at that site, which change sequence. mechanism can be utilized either knock out or introduce selected genes. Given how rapidly gene systems are influencing diverse applications biomedical research, biotechnology, agriculture,1 clinical neurology mutations causing disease, incorporate genes might therapeutic benefit, on near horizon. following cases illustrate such approach may used develop new ex vivo (case 1) 2) therapies for neurologic respectively, highlight some translational challenges using these molecular reagents safely ethically patients.