Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy
EY Osman, PF Yen, CL Lorson - Molecular Therapy, 2012 - cell.com
Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by loss of survival
motor neuron-1 (SMN1). A nearly identical copy gene, SMN2, is present in all SMA patients …
motor neuron-1 (SMN1). A nearly identical copy gene, SMN2, is present in all SMA patients …
Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy
TD Baughan, A Dickson, EY Osman… - Human molecular …, 2009 - academic.oup.com
Spinal muscular atrophy (SMA) is a motor neuron disease caused by the loss of survival
motor neuron-1 (SMN1). A nearly identical copy gene, SMN2, is present in all SMA patients …
motor neuron-1 (SMN1). A nearly identical copy gene, SMN2, is present in all SMA patients …
RNA in spinal muscular atrophy: therapeutic implications of targeting
Introduction Spinal muscular atrophy (SMA) is caused by low levels of the Survival Motor
Neuron (SMN) protein due to deletions of or mutations in the SMN1 gene. Humans carry …
Neuron (SMN) protein due to deletions of or mutations in the SMN1 gene. Humans carry …
Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is caused by loss of the Survival Motor Neuron 1 (SMN1)
gene, resulting in reduced SMN protein. Humans possess the additional SMN2 gene (or …
gene, resulting in reduced SMN protein. Humans possess the additional SMN2 gene (or …
Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy
JH Williams, RC Schray, CA Patterson… - Journal of …, 2009 - Soc Neuroscience
Spinal muscular atrophy (SMA) is caused by homozygous mutation or deletion of the SMN1
gene encoding survival of motor neuron (SMN) protein, resulting in the selective loss of α …
gene encoding survival of motor neuron (SMN) protein, resulting in the selective loss of α …
[HTML][HTML] Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 …
JF Staropoli, H Li, SJ Chun, N Allaire, P Cullen, A Thai… - Genomics, 2015 - Elsevier
Spinal muscular atrophy (SMA) is a neuromuscular disease caused by disruption of the
survival motor neuron 1 (SMN1) gene, partly compensated for by the paralogous gene …
survival motor neuron 1 (SMN1) gene, partly compensated for by the paralogous gene …
Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAs
T Geib, KJ Hertel - PloS one, 2009 - journals.plos.org
Background Spinal Muscular Atrophy (SMA) is an autosomal recessive disease that leads to
specific loss of motor neurons. It is caused by deletions or mutations of the survival of motor …
specific loss of motor neurons. It is caused by deletions or mutations of the survival of motor …
The antisense transcript SMN-AS1 regulates SMN expression and is a novel therapeutic target for spinal muscular atrophy
C d'Ydewalle, DM Ramos, NJ Pyles, SY Ng, M Gorz… - Neuron, 2017 - cell.com
The neuromuscular disorder spinal muscular atrophy (SMA), the most common inherited
killer of infants, is caused by insufficient expression of survival motor neuron (SMN) protein …
killer of infants, is caused by insufficient expression of survival motor neuron (SMN) protein …
Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation
K Meyer, J Marquis, J Trüb… - Human molecular …, 2009 - academic.oup.com
In spinal muscular atrophy (SMA), the leading genetic cause of early childhood death, the
survival motor neuron 1 gene (SMN1) is deleted or inactivated. The nearly identical SMN2 …
survival motor neuron 1 gene (SMN1) is deleted or inactivated. The nearly identical SMN2 …
Antisense oligonucleotide mediated therapy of spinal muscular atrophy
S Sivanesan, MD Howell, CJ DiDonato… - Translational …, 2013 - Springer
Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality. SMA results
from deletions or mutations of survival motor neuron 1 (SMN1), an essential gene. SMN2, a …
from deletions or mutations of survival motor neuron 1 (SMN1), an essential gene. SMN2, a …