Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing.
Odermatt, Philipp
Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing. [electronic resource] - Molecular therapy : the journal of the American Society of Gene Therapy 10 2016 - 1797-1805 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
1525-0024
10.1038/mt.2016.152 doi
Adenoviridae--genetics
Animals
Disease Models, Animal
Genetic Therapy--methods
Genetic Vectors--administration & dosage
Liver--metabolism
Mice
Mice, Transgenic
Muscular Atrophy, Spinal--genetics
Myocardium--metabolism
RNA Splicing
RNA, Small Nuclear--administration & dosage
Survival of Motor Neuron 2 Protein--genetics
Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing. [electronic resource] - Molecular therapy : the journal of the American Society of Gene Therapy 10 2016 - 1797-1805 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
1525-0024
10.1038/mt.2016.152 doi
Adenoviridae--genetics
Animals
Disease Models, Animal
Genetic Therapy--methods
Genetic Vectors--administration & dosage
Liver--metabolism
Mice
Mice, Transgenic
Muscular Atrophy, Spinal--genetics
Myocardium--metabolism
RNA Splicing
RNA, Small Nuclear--administration & dosage
Survival of Motor Neuron 2 Protein--genetics