Immunodeficiency: Gene therapy for primary immune deficiency.

Current gene therapy for inborn errors of immunity lia-ue involved the use of gene addition approaches with -viral delivery. This main strategy has had demonstrated sitccess maility in severe conibilied iminitile deficieng, Wiskott-Aldrich syndrome, and chronic gramiloniatous disease. Despite the increasing success of gene tlierapy, there are limitations of gene therapy, and, therefore, hematopoietic steni cell transplantation continues to be the preferred option. With improvements iii viral delivery through next-generation lentiuiral vectors aild the advent of getle editing zoith CRISPR-Cas9, the efficticy mid safety of gene therapy may soon surpass hematopoietic stem cell tronsplaiitation. 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 liinitations, gene therapy for inborn errors of inimunity is poised to continue to expand to more patients and indications. [ABSTRACT FROM AUTHOR]

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