The clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated
protein 9 (Cas9) system has become a successful and promising technology for gene …

The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …

Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

Inherited retinal diseases (IRDs) are a clinically complex and heterogenous group of visual
impairment phenotypes caused by pathogenic variants in at least 277 nuclear and …

Gene therapy by expression constructs or down-regulation of certain genes has shown great
potential for the treatment of various diseases. The wide clinical application of nucleic acid …

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas
(CRISPR-associated) genome editing systems have become one of the most robust …

CRISPR/Cas9 has become a powerful method for making changes to the genome of many
organisms. First discovered in bacteria as part of an adaptive immune system …