Gene and cell therapies have shown tremendous advancement in the last 5 years. Prominent examples include the successful use of CRISPR-edited stem cells for treating blood disorders like sickle cell anemia and beta-thalassemia, and ongoing clinical trials for treating blindness. This mini-review assesses the status of CRISPR-based therapies, both in vivo and ex vivo, and the challenges associated with clinical translation. In vivo CRISPR therapies have been used to treat eye and liver diseases due to the practicality of delivering editing components to the target tissue. In contrast, even though ex vivo CRISPR therapy involves cell isolation, expansion, and infusion, its advantages include characterizing the gene edits before infusion and restricting off-target effects in other tissues. Further, the safety, affordability, and feasibility of CRISPR therapies, especially for treating large number of patients, are discussed.
Keyphrases
- crispr cas
- genome editing
- genome wide
- stem cells
- dna methylation
- clinical trial
- cell therapy
- single cell
- end stage renal disease
- copy number
- chronic kidney disease
- low dose
- gene expression
- ejection fraction
- prognostic factors
- study protocol
- sickle cell disease
- patient reported outcomes
- iron deficiency
- patient reported