DcpS as a Therapeutic Target for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is caused by deletion or mutation of both copies of the SMN1 gene, which produces an essential protein known as SMN. The severity of SMA is modified by variable copy number of a second gene, SMN2, which produces an mRNA that is incorrectly spliced with deletion of the l...

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Veröffentlicht in:	ACS chemical biology 2008-11, Vol.3 (11), p.711-722
Hauptverfasser:	Singh, Jasbir, Salcius, Michael, Liu, Shin-Wu, Staker, Bart L, Mishra, Rama, Thurmond, John, Michaud, Gregory, Mattoon, Dawn R, Printen, John, Christensen, Jeffery, Bjornsson, Jon Mar, Pollok, Brian A, Kiledjian, Megerditch, Stewart, Lance, Jarecki, Jill, Gurney, Mark E
Format:	Artikel
Sprache:	eng
Schlagworte:	Drug Delivery Systems Endoribonucleases - antagonists & inhibitors Humans Muscular Atrophy, Spinal - drug therapy Protein Binding Protein Conformation - drug effects Quinazolines - pharmacology
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