Ontology highlight
ABSTRACT:
SUBMITTER: Chew J
PROVIDER: S-EPMC4692360 | biostudies-literature | 2015 Jun
REPOSITORIES: biostudies-literature
Chew Jeannie J Gendron Tania F TF Prudencio Mercedes M Sasaguri Hiroki H Zhang Yong-Jie YJ Castanedes-Casey Monica M Lee Chris W CW Jansen-West Karen K Kurti Aishe A Murray Melissa E ME Bieniek Kevin F KF Bauer Peter O PO Whitelaw Ena C EC Rousseau Linda L Stankowski Jeannette N JN Stetler Caroline C Daughrity Lillian M LM Perkerson Emilie A EA Desaro Pamela P Johnston Amelia A Overstreet Karen K Edbauer Dieter D Rademakers Rosa R Boylan Kevin B KB Dickson Dennis W DW Fryer John D JD Petrucelli Leonard L
Science (New York, N.Y.) 20150514 6239
The major genetic cause of frontotemporal dementia and amyotrophic lateral sclerosis is a G4C2 repeat expansion in C9ORF72. Efforts to combat neurodegeneration associated with "c9FTD/ALS" are hindered by a lack of animal models recapitulating disease features. We developed a mouse model to mimic both neuropathological and clinical c9FTD/ALS phenotypes. We expressed (G4C2)66 throughout the murine central nervous system by means of somatic brain transgenesis mediated by adeno-associated virus. Bra ...[more]