Retinal degenerative diseases are a major cause of blindness.Retinal gene therapy is a trail-blazer in the human gene therapy field,leading to the frst FDA approved gene therapy product for a human genetic disease.The...Retinal degenerative diseases are a major cause of blindness.Retinal gene therapy is a trail-blazer in the human gene therapy field,leading to the frst FDA approved gene therapy product for a human genetic disease.The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9(CRISPR/Cas9)-mediated gene editing technology is transforming the delivery of gene therapy.We review the history,present,and future pro-spects of retinal gene therapy.展开更多
文摘Retinal degenerative diseases are a major cause of blindness.Retinal gene therapy is a trail-blazer in the human gene therapy field,leading to the frst FDA approved gene therapy product for a human genetic disease.The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9(CRISPR/Cas9)-mediated gene editing technology is transforming the delivery of gene therapy.We review the history,present,and future pro-spects of retinal gene therapy.
