1.0 NAPNAP contact hour of which 1.0 contain pharmacology content. This continuing education activity is offered free to NAPNAP Members. For nonmembers, this continuing education activity rate is $10. Sickle cell disease (SCD) is a lifelong disease requiring expensive treatment for management and limited curative options until the last few years. Gene therapy has emerged as a curative option for SCD, with two approved therapies available to SCD patients aged greater than 12 years. Consideration must be considered regarding the ethics, efficacy, management requirements, education, and counseling needs of patients and their parents. Current and future practices will need to advocate for improved access and affordability of this specialized care and address the unknown and less defined areas of gene therapy pertaining to SCD through research.
J Pediatr Health Care. (2025) 39, Issue 1, 122-128.

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Copyright © 2025 by the National Association of Pediatric Nurse Practitioners. Published by Elsevier Inc. All rights reserved.

This continuing education activity is administered by the National Association of Pediatric Nurse Practitioners (NAPNAP) as an Agency providing continuing education credit. Individuals who complete this program and earn a 70% or higher score on the post test will be awarded 1.0 NAPNAP contact hour of which 1.0 contain pharmacology content.

Keywords: Gene therapy, sickle cell disease, adolescence, CRISPER, lentiviral vector