Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy

202260 citationsReviewgreen Open Access

Authors

Ashish O. Gupta · University of Minnesota

Gerald V. Raymond · Kennedy Krieger Institute

Elizabeth I. Pierpont · University of Minnesota

Stephan Kemp · Amsterdam Neuroscience

R. Scott McIvor · University of Minnesota

Arpana Rayannavar · University of Minnesota

Bradley S. Miller ·

Abstract

The data regarding this lentiviral-based gene therapy approach and its relative risks and benefits is still being evaluated. This information is explored in the context of the experience with allogeneic HSCT for cerebral ALD.

Topics & Keywords

Peroxisome Proliferator-Activated Receptors Virus-based gene therapy research Metabolism and Genetic Disorders

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Expert Opinion on Biological Therapy

Volume 22, Issue 9, pp. 1151-1162

DOI: 10.1080/14712598.2022.2124857

Field-Weighted Citation Impact: 5.03