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Research Article
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Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy

Anne Rietz, Kevin J Hodgetts, Hrvoje Lusic, Kevin M Quist, View ORCID ProfileErkan Y Osman, View ORCID ProfileChristian L Lorson, View ORCID ProfileElliot J Androphy  Correspondence email
Anne Rietz
1Department of Dermatology, Indiana University School of Medicine, Indianapolis, IN, USA
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Kevin J Hodgetts
2Laboratory for Drug Discovery in Neurodegeneration, Brigham and Women’s Hospital, Harvard Medical School, Cambridge, MA, USA
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Hrvoje Lusic
2Laboratory for Drug Discovery in Neurodegeneration, Brigham and Women’s Hospital, Harvard Medical School, Cambridge, MA, USA
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Kevin M Quist
1Department of Dermatology, Indiana University School of Medicine, Indianapolis, IN, USA
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Erkan Y Osman
3Department of Veterinary Pathobiology, Bond Life Sciences Center, College of Veterinary Medicine, University of Missouri, Columbia, MO, USA
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  • ORCID record for Erkan Y Osman
Christian L Lorson
3Department of Veterinary Pathobiology, Bond Life Sciences Center, College of Veterinary Medicine, University of Missouri, Columbia, MO, USA
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  • ORCID record for Christian L Lorson
Elliot J Androphy
1Department of Dermatology, Indiana University School of Medicine, Indianapolis, IN, USA
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  • ORCID record for Elliot J Androphy
  • For correspondence: eandro@iu.edu
Published 24 November 2020. DOI: 10.26508/lsa.202000889
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A pharmacologically tunable SMA mouse model
Anne Rietz, Kevin J Hodgetts, Hrvoje Lusic, Kevin M Quist, Erkan Y Osman, Christian L Lorson, Elliot J Androphy
Life Science Alliance Nov 2020, 4 (1) e202000889; DOI: 10.26508/lsa.202000889

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A pharmacologically tunable SMA mouse model
Anne Rietz, Kevin J Hodgetts, Hrvoje Lusic, Kevin M Quist, Erkan Y Osman, Christian L Lorson, Elliot J Androphy
Life Science Alliance Nov 2020, 4 (1) e202000889; DOI: 10.26508/lsa.202000889
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Volume 4, No. 1
January 2021
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  • Genetics, Gene Therapy & Genetic Disease
  • Neuroscience

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