1
40
1
-
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
June 2021 List
Text
A resource consisting primarily of words for reading. Examples include books, letters, dissertations, poems, newspapers, articles, archives of mailing lists. Note that facsimiles or images of texts are still of the genre Text.
Citation List Month
June 2021 List
URL Address
<a href="http://doi.org/10.1111/dmcn.14885" target="_blank" rel="noreferrer noopener">http://doi.org/10.1111/dmcn.14885</a>
Dublin Core
The Dublin Core metadata element set is common to all Omeka records, including items, files, and collections. For more information see, http://dublincore.org/documents/dces/.
Title
A name given to the resource
Viral gene therapy for paediatric neurological diseases: progress to clinical reality
Publisher
An entity responsible for making the resource available
Developmental Medicine & Child Neurology
Date
A point or period of time associated with an event in the lifecycle of the resource
2021
Subject
The topic of the resource
clinical; life-limiting; neurological diseases; paediatric; viral gene therapy
Creator
An entity primarily responsible for making the resource
Privolizzi R; Chu WS; Tijani M; Ng J
Description
An account of the resource
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.
Identifier
An unambiguous reference to the resource within a given context
<a href="http://doi.org/10.1111/dmcn.14885" target="_blank" rel="noreferrer noopener">10.1111/dmcn.14885</a>
Rights
Information about rights held in and over the resource
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).
2021
Chu WS
Clinical
Developmental Medicine & Child Neurology
June 2021 List
Life-limiting
neurological diseases
Ng J
Paediatric
Privolizzi R
Tijani M
viral gene therapy