RT Journal Article
T1 Current developments in gene therapy for amyotrophic lateral sclerosis
A1 Scarrott, Joseph M.
A1 Herranz Martín, Saúl
A1 Alrafiah, Aziza R.
A1 Shaw, Pamela J.
A1 Azzouz, Mimoun
AB Introduction: Amyotrophic lateral sclerosis (ALS) is a devastating adult neurodegenerative disorder characterized by motor neuron degeneration and death around 3 years from onset. So far, riluzole is the only treatment available, although it only offers a slight increase in survival. The complex etiology of ALS, with several genes able to trigger the disease, makes its study difficult.Areas covered: RNA-mediated or protein-mediated toxic gain-of-function leading to motor neuron degeneration appears to be likely common pathogenic mechanisms in ALS. Consequently, gene therapy technologies to reduce toxic RNA and/or proteins and to protect motor neurons by modulating gene expression are at the forefront of the field. Here, we review the most promising scientific advances, paying special attention to the successful treatments tested in animal models as well as analyzing relevant gene therapy clinical trials.Expert opinion: Despite broad advances in target gene identification in ALS and advances in gene therapy technologies, a successful gene therapy for ALS continues to elude researchers. Multiple hurdles encompassing technical, biological, economical and clinical challenges must be overcome before a therapy for patients becomes available. Optimism remains due to positive results obtained in several in vivo studies demonstrating significant disease amelioration in animal models of ALS.
PB Taylor and Francis Group
SN 1471-2598
SN 1744-7682
YR 2015
FD 2015-05-10
LK https://hdl.handle.net/20.500.14352/96283
UL https://hdl.handle.net/20.500.14352/96283
LA eng
NO Scarrott JM, Herranz-Martín S, Alrafiah AR, Shaw PJ, Azzouz M. Current developments in gene therapy for amyotrophic lateral sclerosis. Expert Opin Biol Ther. 2015 Jul;15(7):935-47. doi: 10.1517/14712598.2015.1044894. Epub 2015 May 10. PMID: 25959569.
DS Docta Complutense
RD 28 abr 2025