Viral gene therapy for paediatric neurological diseases: progress to clinical reality

Title

Viral gene therapy for paediatric neurological diseases: progress to clinical reality

Creator

Privolizzi R; Chu WS; Tijani M; Ng J

Identifier

Publisher

Developmental Medicine & Child Neurology

Date

2021

Subject

clinical; life-limiting; neurological diseases; paediatric; viral gene therapy

Description

In the era of genomic medicine, diagnoses of rare paediatric neurological diseases are increasing. Many are untreatable and life-limiting, leading to an exceptional increase in gene therapy development. It is estimated that 20 gene therapy products will have received approval from the US Food and Drug Administration by 2025. With viral gene therapy considered a potential single-dose cure for patients with spinal muscular atrophy type 1 as one example, and contemporaneously tragically resulting in the deaths of three male children with X-linked myotubular myopathy receiving high-dose gene therapy in 2020, what is the current state of gene therapy? What is behind the decades of hype around viral gene therapy and is it high impact, but high risk? In this review, we outline principles of viral gene therapy development and summarize the most recent clinical evidence for the therapeutic effect of gene therapy in paediatric neurological diseases. We discuss adeno-associated virus and lentiviral vectors, antisense oligonucleotides, emerging genetic editing approaches, and current limitations that the field still faces.

Rights

Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).

Citation List Month

June 2021 List

Collection

Citation

Privolizzi R; Chu WS; Tijani M; Ng J, “Viral gene therapy for paediatric neurological diseases: progress to clinical reality,” Pediatric Palliative Care Library, accessed July 26, 2021, https://pedpalascnetlibrary.omeka.net/items/show/17581.

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