Gene Therapy for Sickle Cell Disease

Abstract

Background:

Sickle cell disease (SCD) is a genetic blood disorder in which sickled red blood cells cannot easily pass through the vasculature, leading to impaired tissue perfusion and oxygenation that can lead to vaso-occlusive pain, stroke, and multiorgan and tissue damage. Scientists have now developed genetic approaches to treating SCD with a curative intent. The purpose of this article is to inform the reader of the current state of SCD gene therapy and an institution's experience and outcomes with this therapy.

Method:

Gene therapy uses the patient's stem cells to create a patient-specific genetically modified product to correct or replace the genetic mutation causing the SCD. The two genetic treatment approaches to SCD gene therapy are gene addition/gene transfer and gene editing. Patients experience a multistep, complex process to undergo this treatment, requiring close communication between the patient and the interdisciplinary teams at the patient's home treatment center and gene therapy treatment site. Phase I, II, and III clinical trials have been completed or are ongoing worldwide.

Results:

Based on clinical trial findings, in 2023, the Food and Drug Administration approved two gene therapy products. At one treatment site, 17 patients have completed treatment on two clinical trials, and all are alive and stable without serious adverse events related to the gene therapy product.

Discussion:

As gene therapy treatments continue to develop as an SCD treatment option, nurses play an important role in educating patients and their families about the therapy. Patients undergoing gene therapy need close nursing care coordination and expert supportive care during all steps of the treatment process, including follow-up.