The development Clustered Regularly Interspaced Short Palindromic Repeats-Cas (CRISPR-Cas) genome editing technology has made an unconceivable impact in the world of medicine. CRISPR/Cas technology has potential precisely target the source of the disease and eliminate it with as little as a single dose of drug. It has been the last hope in improving the quality of life for many patients suffering from genetic disorders such as Duchenne muscular dystrophy (DMD) or diseases with undruggable targets such as Non-alcoholic steatohepatitis(NASH).
CRISPR/Cas technology in the clinical trials is currently limited to treating monogenic diseases of blood or liver origin. The requirement of a specific Protospacer Adjacent Motif (PAM) sequence to target a particular sequence of interest greatly limits our potential to target a wide variety of genes and the unavailability of gene delivery technologies to brain and lung are the major limitations in using CRISPR/Cas to treat diseases of the brain or lung. To advance the employability of CRISPR/Cas technology for treating a widespread of diseases in the clinic, discovering new Cas proteins with flexible PAM constraints, development of novel gene/protein delivery technologies, as well as engineering new variants of CRISPR/Cas technologies are the key.
The aim of the current Research Topic is to cover promising, recent, and novel research and review trends in the genome editing field. Areas to be covered in this Research Topic may include, but are not limited to:
• Advances and current issues with delivery of CRISPR/Cas technologies at a molecular and mechanistic level
• Advances and current issues with the development of novel CRISPR/Cas technologies at a molecular level
The development Clustered Regularly Interspaced Short Palindromic Repeats-Cas (CRISPR-Cas) genome editing technology has made an unconceivable impact in the world of medicine. CRISPR/Cas technology has potential precisely target the source of the disease and eliminate it with as little as a single dose of drug. It has been the last hope in improving the quality of life for many patients suffering from genetic disorders such as Duchenne muscular dystrophy (DMD) or diseases with undruggable targets such as Non-alcoholic steatohepatitis(NASH).
CRISPR/Cas technology in the clinical trials is currently limited to treating monogenic diseases of blood or liver origin. The requirement of a specific Protospacer Adjacent Motif (PAM) sequence to target a particular sequence of interest greatly limits our potential to target a wide variety of genes and the unavailability of gene delivery technologies to brain and lung are the major limitations in using CRISPR/Cas to treat diseases of the brain or lung. To advance the employability of CRISPR/Cas technology for treating a widespread of diseases in the clinic, discovering new Cas proteins with flexible PAM constraints, development of novel gene/protein delivery technologies, as well as engineering new variants of CRISPR/Cas technologies are the key.
The aim of the current Research Topic is to cover promising, recent, and novel research and review trends in the genome editing field. Areas to be covered in this Research Topic may include, but are not limited to:
• Advances and current issues with delivery of CRISPR/Cas technologies at a molecular and mechanistic level
• Advances and current issues with the development of novel CRISPR/Cas technologies at a molecular level
