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Search Results - Frair, Emma C
Search Results - Frair, Emma C
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Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
by
Wein, Nicolas
,
Vetter, Tatyana A.
,
Vulin, Adeline
,
Simmons, Tabatha R.
,
Frair, Emma C.
,
Bradley, Adrienne J.
,
Gushchina, Liubov V.
,
Almeida, Camila F.
,
Huang, Nianyuan
,
Lesman, Daniel
,
Rajakumar, Dhanarajan
,
Weiss, Robert B.
,
Flanigan, Kevin M.
Published in
Molecular therapy. Methods & clinical development
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Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy
by
Gushchina, Liubov V.
,
Vetter, Tatyana A.
,
Frair, Emma C.
,
Bradley, Adrienne J.
,
Grounds, Kelly M.
,
Lay, Jacob W.
,
Huang, Nianyuan
,
Suhaiba, Aisha
,
Schnell, Frederick J.
,
Hanson, Gunnar
,
Simmons, Tabatha R.
,
Wein, Nicolas
,
Flanigan, Kevin M.
Published in
Molecular therapy. Nucleic acids
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Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
by
Gushchina, Liubov V.
,
Bradley, Adrienne J.
,
Vetter, Tatyana A.
,
Lay, Jacob W.
,
Rohan, Natalie L.
,
Frair, Emma C.
,
Wein, Nicolas
,
Flanigan, Kevin M.
Published in
Molecular therapy. Methods & clinical development
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CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice
by
Stephenson, Anthony A.
,
Nicolau, Stefan
,
Vetter, Tatyana A.
,
Dufresne, Gabrielle P.
,
Frair, Emma C.
,
Sarff, Jessica E.
,
Wheeler, Gregory L.
,
Kelly, Benjamin J.
,
White, Peter
,
Flanigan, Kevin M.
Published in
Molecular therapy. Methods & clinical development
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A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2
by
Flanigan, Kevin M.
,
Vetter, Tatyana A.
,
Simmons, Tabatha R.
,
Iammarino, Megan
,
Frair, Emma C.
,
Rinaldi, Federica
,
Chicoine, Louis G.
,
Harris, Johan
,
Cheatham, John P.
,
Cheatham, Sharon L.
,
Boe, Brian
,
Waldrop, Megan A.
,
Zygmunt, Deborah A.
,
Packer, Davin
,
Martin, Paul T.
Published in
Molecular therapy. Methods & clinical development
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Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies
by
Vetter, Tatyana A.
,
Nicolau, Stefan
,
Bradley, Adrienne J.
,
Frair, Emma C.
,
Flanigan, Kevin M.
Published in
Neuropathology and applied neurobiology
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A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK. GALGT2
by
Flanigan, Kevin M
,
Vetter, Tatyana A
,
Simmons, Tabatha R
,
Iammarino, Megan
,
Frair, Emma C
,
Rinaldi, Federica
,
Chicoine, Louis G
,
Harris, Johan
,
Cheatham, John P
,
Cheatham, Sharon L
,
Boe, Brian
,
Waldrop, Megan A
,
Zygmunt, Deborah A
,
Packer, Davin
,
Martin, Paul T
Published in
Molecular therapy. Methods & clinical development
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Peer Reviewed
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Journal Title
Molecular Therapy. Methods & Clinical Development
5 results
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Molecular Therapy. Nucleic Acids
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Neuropathology & Applied Neurobiology/Neuropathology And Applied Neurobiology
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Neuropathology And Applied Neurobiology
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Life Sciences & Biomedicine
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Science & Technology
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Duchenne Muscular Dystrophy
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Dystrophin
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Medicine, Research & Experimental
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Research & Experimental Medicine
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Gene Therapy
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Becker Muscular Dystrophy
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Original
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Exon Skipping
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U7Snrna
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Aav
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Antisense Oligonucleotide, Ao
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Automation
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Biopsy
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