The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.

Gogliotti, Rocky G; Cardona, Herminio; Singh, Jasbir; Bail, Sophie; Emery, Carina; Kuntz, Nancy; Jorgensen, Michael; Durens, Madel; Xia, Bing; Barlow, Courtenay; Heier, Christopher R; Plasterer, Heather L; Jacques, Vincent; Kiledjian, Megerditch; Jarecki, Jill; Rusche, James; DiDonato, Christine J

Gogliotti, Rocky G; Cardona, Herminio; Singh, Jasbir; Bail, Sophie; Emery, Carina; Kuntz, Nancy; Jorgensen, Michael; Durens, Madel; Xia, Bing; Barlow, Courtenay; Heier, Christopher R; Plasterer, Heather L; Jacques, Vincent; Kiledjian, Megerditch; Jarecki, Jill; Rusche, James; DiDonato, Christine J.

Affiliation

Gogliotti RG; These authors contributed equally to this study.

Hum Mol Genet ; 22(20): 4084-101, 2013 Oct 15.

Article in En | MEDLINE | ID: mdl-23736298

Subject(s)

Endoribonucleases/antagonists & inhibitors; Motor Neurons/drug effects; Motor Neurons/pathology; Muscular Atrophy, Spinal/physiopathology; Quinazolines/pharmacology; Quinazolines/pharmacokinetics; Survival of Motor Neuron 2 Protein/metabolism; Administration, Oral; Animals; Disease Models, Animal; Dose-Response Relationship, Drug; Drug Evaluation, Preclinical; Endoribonucleases/metabolism; Female; Humans; Mice; Mice, Transgenic; Muscular Atrophy, Spinal/drug therapy; Quinazolines/therapeutic use; Survival of Motor Neuron 2 Protein/genetics; Synapses/drug effects; Synapses/physiology

Fulltext

XML

PubMed Links

Search on Google

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Quinazolines / Muscular Atrophy, Spinal / Endoribonucleases / Survival of Motor Neuron 2 Protein / Motor Neurons Limits: Animals / Female / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2013 Type: Article

Fulltext

XML

PubMed Links

Search on Google