TY  - GEN
AU  - Odermatt,Philipp
AU  - Trüb,Judith
AU  - Furrer,Lavinia
AU  - Fricker,Roger
AU  - Marti,Andreas
AU  - Schümperli,Daniel
TI  - Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing
SN  - 1525-0024
PY  - 2017///0622
KW  - Adenoviridae
KW  - genetics
KW  - Animals
KW  - Disease Models, Animal
KW  - Genetic Therapy
KW  - methods
KW  - Genetic Vectors
KW  - administration & dosage
KW  - Liver
KW  - metabolism
KW  - Mice
KW  - Mice, Transgenic
KW  - Muscular Atrophy, Spinal
KW  - Myocardium
KW  - RNA Splicing
KW  - RNA, Small Nuclear
KW  - Survival of Motor Neuron 2 Protein
N1  - Publication Type: Journal Article; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1038/mt.2016.152
ER  -