Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.

Autor:	McMillan HJ; Departments of Pediatrics, Neurology & Neurosurgery, Montreal Children's Hospital, McGill University Health Centre, Montreal, Canada., Proud CM; Children's Hospital of the King's Daughters, Norfolk, VA, United States., Farrar MA; School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.; Department of Neurology, Sydney Children's Hospital Network, Sydney, NSW, Australia., Alexander IE; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW, Australia.; Discipline of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW, Australia., Muntoni F; The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health London, UK.; NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, UK., Servais L; Department of Pediatrics, Centre Hospitalier Universitaire de Liège & Université de Liège, Liège, Belgium.; MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, UK.
Jazyk:	angličtina
Zdroj:	Expert opinion on biological therapy [Expert Opin Biol Ther] 2022 Sep; Vol. 22 (9), pp. 1075-1090. Date of Electronic Publication: 2022 May 02.
DOI:	10.1080/14712598.2022.2066471
Abstrakt:	Introduction: Gene therapy for spinal muscular atrophy (SMA) represents a significant milestone in the treatment of neurologic diseases. SMA is a neurodegenerative disease that results in motor neuron loss because of mutations of the survival motor neuron 1 gene, which directs survival motor neuron (SMN) protein production. Onasemnogene abeparvovec, a one-time gene replacement therapy, delivers a functional transgene to restore SMN protein expression. Onasemnogene abeparvovec has demonstrated improved survival and motor milestone achievements for presymptomatic infants and patients with SMA type 1. Areas Covered: This expert review describes the current state of gene therapy for SMA, reviews the mechanism of and clinical experience with onasemnogene abeparvovec, explains future efforts to expand applications of gene therapy for SMA, and provides context for developing gene therapy for other conditions. Expert Opinion: Onasemnogene abeparvovec has demonstrated efficacy in clinical trials and, because of this, is a valuable treatment option for patients with symptomatic infantile SMA and those identified by newborn screening. Gene therapy is still in its infancy, and challenges and uncertainties associated with transgene delivery must be addressed. With ongoing development of vector technology, more specific tissue tropism, reduced 'off-target' effects, and an enhanced safety profile will continue to evolve.
Databáze:	MEDLINE
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