Gene therapy to treat mucopolysaccharidosis IVA

Reporting in the peer-reviewed journal Human Gene Therapy, researchers used ex vivo lentiviral gene therapy to treat mucopolysaccharidosis IVA (MPS IVA) in mice. Click here to read the article now.

MPS IVA is an autosomal recessive disease caused by a mutation in the N-acetylgalactosamine-5-sulfate sulfatase (GALNS) gene resulting in progressive system skeletal dysplasia. The goal of the ex vivo gene therapy is to transduce hemtopoietic stem cells (HSCs) with lentivirus  (LV) carrying the GALNS gene under two different promoters, thereby producing supraphysiological levels of active GALNS enzyme.

Shunji Tomatsu, from Nemours Children’s Health, and coauthors, reported that LV-HSC gene therapy did not reach the normal physiological level of the GLANS enzyme, suggesting that further efforts to optimize the therapy  such as improved LVs or enhanced conditioning may be required to achieve supraphysiological levels.

“This study has unveiled a promising potential for the treatment of MPS IVA,” concluded the investigators. “We observed a partical correction of bone pathology and a complete correction in heart pathology (heart valves and muscle) in the MPS IVA mouse model.”

“New and better medicinces are the result of numerous incremental improvements in the formulation of the vector and optimization of treatment conditions, and this is a positive step forward for developing novel therapies to treat patients with MPS IVA,” says Managing Editor of Human Gene Therapy Thomas Gallagher, PhD, from the University of Massachusetts Chan Medical School.

About the Journal
Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available on the Human Gene Therapy website.

About the Publisher
Mary Ann Liebert, Inc. is a global media company dedicated to creating, curating, and delivering impactful peer-reviewed research and authoritative content services to advance the fields of biotechnology and the life sciences, specialized clinical medicine, and public health and policy. For complete information, please visit the Mary Ann Liebert, Inc. website.