z-logo
Premium
Modern approaches in gene therapy of motor neuron diseases
Author(s) -
Zakharova Maria
Publication year - 2021
Publication title -
medicinal research reviews
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.868
H-Index - 130
eISSN - 1098-1128
pISSN - 0198-6325
DOI - 10.1002/med.21705
Subject(s) - spinal muscular atrophy , amyotrophic lateral sclerosis , sma* , motor neuron , medicine , genetic enhancement , neuroscience , weakness , atrophy , bioinformatics , progressive muscular atrophy , physical medicine and rehabilitation , disease , gene , pathology , biology , genetics , surgery , mathematics , combinatorics
Abstract Motor neuron disorders are a group of neurodegenerative diseases characterized by muscle weakness, loss of ambulation, respiratory insufficiency, leading to an early death. Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis are the most common and fatal motor neuron diseases. The last 3 years became very successful for novel gene therapy approaches in SMA in infants. Two innovative drugs—nusinersen (Spinraza) and onasemnogene abeparvovec (Zolgensma) have been approved by health authorities. The numerous molecular and genetic overlaps between different neurodegenerative diseases are of great importance in the development of innovative therapeutic strategies, including viral vector therapy and RNA modulating approaches.

This content is not available in your region!

Continue researching here.

Having issues? You can contact us here