Nusinersen in the Treatment of Spinal Muscular Atrophy

Methods in Molecular Biology
Kara GoodkeyToshifumi Yokota

Abstract

Spinal muscular atrophy (SMA) is one of the most common genetic causes of infantile death arising due to mutations in the SMN1 gene and the subsequent loss of motor neurons. With the discovery of the intronic splicing silencer N1 (ISS-N1) as a potential target for antisense therapy, several antisense oligonucleotides (ASOs) are being developed to include exon 7 in the final mRNA transcript of the SMN2 gene and thereby increasing the production of spinal motor neuron (SMN) proteins. Nusinersen (spinraza), a modified 2'-O-methoxyethyl (MOE) antisense oligonucleotide is the first drug to be approved by Food and Drug Agency (FDA) in December of 2016. Here we briefly review the pharmacological relevance of the drug, clinical trials, toxicity, and future directions following the approval of nusinersen.

Citations

Jun 6, 2019·Nucleic Acids Research·Xue-Hai LiangStanley T Crooke
Jun 23, 2019·Human Molecular Genetics·Bhavya RaviAndrew P Lieberman
Sep 15, 2019·Indian Journal of Pediatrics·Nagehan AslanNeslihan Ozcan
Feb 8, 2020·Journal of Cellular and Molecular Medicine·Irene FaravelliAlessandra Govoni
Jun 27, 2020·Mass Spectrometry Reviews·James Michael SuttonMichael G Bartlett
Jul 15, 2020·Chembiochem : a European Journal of Chemical Biology·Melissa WojtyniakChristian Ducho
Oct 8, 2020·Nucleic Acid Therapeutics·Suxiang ChenRakesh N Veedu

❮ Previous
Next ❯

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Antisense Oligonucleotides: ND

This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases including amyotrophic lateral sclerosis.

Cajal Bodies & Gems

Cajal bodies or coiled bodies are dense foci of coilin protein. Gemini of Cajal bodies, or gems, are microscopically similar to Cajal bodies. It is believed that Cajal bodies play important roles in RNA processing while gems assist the Cajal bodies. Find the latest research on Cajal bodies and gems here.