CRISPR/Cas9?system?and Gene editing therapy

Gene therapy, to be brief, is delivering corrective gene materials into cells to treat or alleviate the symptom of disease [86], so far, including gene editing therapy mediated by CRISPR/Cas sytem, ZFN or TALEN etc., and gene replacement, such as lentivirus/adenovirus/AAV-mediated gene expression [20]. Gene editing therapy via CRISPR technology is to correct the pathogenic mutation into non-pathogenic ones, such as gene silence or disruption by CRISPR mediated knockout, which has been widely used in the therapy of hemophilia B [19], Huntington’s disease [87], Parkinson’s Disease [88], hematologic diseases, infectious diseases and malignant tumor [89]. Gene replacement therapy by lentivirus/adenovirus/AAV is to replace the pathogenic gene with its corrective type, which is widely applied in treating lipoprotein lipase deficiency (LPLD) [90], spinal muscular atrophy (SMA) [91], retinal dystrophy [92,93], cystic fibrosis [94,95] muscular dystrophies [96]. Combined CRISPR with virus vectors, AAV-SaCas9 or lentivirus-Cas9 has been used for gene therapy of Hemophilia A [61], Usher Syndrome [97], Intervertebral Disc Degeneration [98] etc.

Click here for Pipelines landscapes of gene replacement therapy and gene editing therapy of different companies..