Antisense-based therapy for the treatment of spinal muscular atrophy

One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal...

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Veröffentlicht in:	The Journal of cell biology 2012-10, Vol.199 (1), p.21-25
Hauptverfasser:	Rigo, Frank, Hua, Yimin, Krainer, Adrian R, Bennett, C Frank
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Humans Mice Mice, Transgenic Muscular Atrophy, Spinal - drug therapy Muscular Atrophy, Spinal - genetics Oligonucleotides, Antisense - pharmacology RNA, Messenger - drug effects RNA, Messenger - genetics Survival of Motor Neuron 1 Protein - antagonists & inhibitors Survival of Motor Neuron 1 Protein - genetics Survival of Motor Neuron 2 Protein - antagonists & inhibitors Survival of Motor Neuron 2 Protein - genetics
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Beschreibung
Zusammenfassung:	One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that has led to a promising drug based on the biology underlying the disease.
ISSN:	0021-9525 1540-8140
DOI:	10.1083/jcb.201207087