https://onlinelibrary.wiley.com/doi/abs/10.1002/med.21705
https://doi.org/10.1002/med.21705
https://pubmed.ncbi.nlm.nih.gov/32638429/
TITLE:
Modern approaches in gene therapy of motor neuron diseases
ALTERNATIVE TITLE:
None
DATE:
Verify – Wed, 08 Jul 2020 06:00:00 -0400
AUTHORS:
Maria Zakharova
SOURCE:
Medicinal research reviews
DESCRIPTION:
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…
CONTENT:
Zakharova M. Med Res Rev 2020 – Review .
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.
PMID:32638429 | DOI:10.1002/med.21705
PUBMED ID:
pubmed:32638429
OTHER ID:
pmid:32638429,doi:10.1002/med.21705
PUBLICATION DATE:
Wed, 08 Jul 2020 06:00:00 -0400
2020-07-09
RETRIEVAL DATE :
07/08/20 02:24PM
LINK – PUBMED:
https://pubmed.ncbi.nlm.nih.gov/32638429/?utm_source=MS-Office&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1l7lJQBQXfTBJkL86rnvYKzafMiKbgcUrlv_X8_D_H5EuRmkjR&fc=20200708141943&ff=20200708142144&v=2.10.1
LINK – DOI:
https://doi.org/10.1002/med.21705
LINK – FULL TEXT:
Pending
NOTES:
None