Gene Therapy for Childhood Cerebral Adrenoleukodystrophy
Year: 2018
Type: article
Abstract: Cellular therapy with allogeneic hematopoietic cell transplantation can stabilize disease and prevent progression in boys with early stage cerebral adrenoleukodystrophy. Gene therapy using modified autologous hematopoietic stem cells is emerging as a potential therapy and possible replacement for allogeneic transplant for children with cerebral adrenoleukodystrophy (ALD). This article examines the role of cellular therapy, allogeneic transplant and autologous gene therapy in boys with ALD. It highlights the published results of two investigations of gene therapy in this setting that show efficacy comparable to allogeneic transplant. Longer term follow-up is needed to fully understand the impact that this new treatment will have on the clinical care of boys with cerebral ALD. Submitted for peer review: 15 Aug 2018 Published: 16 Oct 2018
Source: Cell and Gene Therapy Insights
Author: Christine Duncan
Language: English
Subfield: Physiology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
OpenAlex ID: W2896242552