Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1.
Malerba, Alberto
Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1. [electronic resource] - Human molecular genetics 10 2019 - 3301-3308 p. digital
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
1460-2083
10.1093/hmg/ddz167 doi
Animals
Dependovirus--genetics
Disease Models, Animal
Gene Knockdown Techniques
Genetic Vectors
Humans
Intranuclear Inclusion Bodies--metabolism
Mice
Mice, Transgenic
Muscular Dystrophy, Oculopharyngeal--genetics
Poly(A)-Binding Protein I--genetics
Trinucleotide Repeat Expansion
Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1. [electronic resource] - Human molecular genetics 10 2019 - 3301-3308 p. digital
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
1460-2083
10.1093/hmg/ddz167 doi
Animals
Dependovirus--genetics
Disease Models, Animal
Gene Knockdown Techniques
Genetic Vectors
Humans
Intranuclear Inclusion Bodies--metabolism
Mice
Mice, Transgenic
Muscular Dystrophy, Oculopharyngeal--genetics
Poly(A)-Binding Protein I--genetics
Trinucleotide Repeat Expansion