Current gene therapy for inborn errors of immunity have involved the use of gene addition approaches with viral delivery. This main strategy has had demonstrated success mainly in severe combined immune deficiency, Wiskott-Aldrich syndrome, and chronic granulomatous disease. Despite the increasing success of gene therapy, there are limitations of gene therapy, and, therefore, hematopoietic stem cell transplantation continues to be the preferred option. With improvements in viral delivery through next-generation lentiviral vectors and the advent of gene editing with CRISPR-Cas9, the efficacy and safety of gene therapy may soon surpass hematopoietic stem cell transplantation. Furthermore, these advances improve the viability of gene therapy for inborn errors of immunity primarily through decreased risk of transplantation-related complications. Therefore, despite current limitations, gene therapy for inborn errors of immunity is poised to continue to expand to more patients and indications.
Keyphrases
- gene therapy
- crispr cas
- copy number
- genome wide
- sars cov
- end stage renal disease
- patient safety
- newly diagnosed
- genome wide identification
- adverse drug
- genome editing
- early onset
- peritoneal dialysis
- prognostic factors
- emergency department
- replacement therapy
- risk factors
- mesenchymal stem cells
- case report
- drug induced
- dna methylation
- quality improvement
- bone marrow
- patient reported