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Abstract
Over the past three decades, gene therapy has made significant strides in treating immunodeficiency diseases. This review article provides an overview of gene therapy's historical development, spanning multiple phase trials and clinical applications. It discusses various delivery vectors, both viral and non-viral, and evaluates their success rates. The article briefly touches on the immune system before delving into primary and secondary immunodeficiency disorders. Specific primary immunodeficiency diseases, such as ADA-SCID, X-SCID, ART-SCID, Wiskott-Aldrich Syndrome, X-CGD, and LAD-1, are explored in terms of their causation, treatment options, with a focus on gene therapy, and the latest advancements in clinical trials. While Strimvelis' success in treating ADA-SCID is highlighted, ongoing research for other PIDs is also discussed, outlining their path to success through various trials. By addressing challenges such as long-term effects, off-target effects, and immune responses to gene therapy treatments, the review provides insight into the complexities of this therapeutic approach. However, it also underscores recent achievements and advancements, illustrating not only the future of gene therapy but also its potential to transform healthcare as a whole.
