AAV9-mediated delivery of miR-23a reduces disease severity in Smn2B/−SMA model mice
Kaifer, Kevin A, Villalón, Eric, O'Brien, Benjamin S, Sison, Samantha L, Smith, Caley E, Simon, Madeline E, Marquez, Jose, O'Day, Siri, Hopkins, Abigail E, Neff, Rachel, Rindt, Hansjörg, Ebert, Allison D, Lorson, Christian L
Published in Human molecular genetics (01.10.2019)
Published in Human molecular genetics (01.10.2019)
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Journal Article
Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases
Kline, Rachel A, Kaifer, Kevin A, Osman, Erkan Y, Carella, Francesco, Tiberi, Ariana, Ross, Jolill, Pennetta, Giuseppa, Lorson, Christian L, Murray, Lyndsay M
Published in PLoS genetics (31.03.2017)
Published in PLoS genetics (31.03.2017)
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Journal Article
The adeno-associated virus 2 genome and Rep 68/78 proteins interact with cellular sites of DNA damage
Boftsi, Maria, Whittle, Fawn B, Wang, Juexin, Shepherd, Phoenix, Burger, Lisa R, Kaifer, Kevin A, Lorson, Christian L, Joshi, Trupti, Pintel, David J, Majumder, Kinjal
Published in Human molecular genetics (21.03.2022)
Published in Human molecular genetics (21.03.2022)
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Journal Article
AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice
Kaifer, Kevin A., Villalón, Eric, Smith, Caley E., Simon, Madeline E., Marquez, Jose, Hopkins, Abigail E., Morcos, Toni I., Lorson, Christian L.
Published in Biochemical and biophysical research communications (10.09.2020)
Published in Biochemical and biophysical research communications (10.09.2020)
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Journal Article
Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy
Osman, Erkan Y, Washington, Charles W, Kaifer, Kevin A, Mazzasette, Chiara, Patitucci, Teresa N, Florea, Kyra M, Simon, Madeline E, Ko, Chien-Ping, Ebert, Allison D, Lorson, Christian L
Published in Molecular therapy (01.09.2016)
Published in Molecular therapy (01.09.2016)
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Journal Article
Functional characterization of SMN evolution in mouse models of SMA
Osman, Erkan Y, Bolding, Madeline R, Villalón, Eric, Kaifer, Kevin A, Lorson, Zachary C, Tisdale, Sarah, Hao, Yue, Conant, Gavin C, Pires, J Chris, Pellizzoni, Livio, Lorson, Christian L
Published in Scientific reports (01.07.2019)
Published in Scientific reports (01.07.2019)
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Journal Article
Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCFSlmb degron
Gray, Kelsey M, Kaifer, Kevin A, Baillat, David, Wen, Ying, Bonacci, Thomas R, Ebert, Allison D, Raimer, Amanda C, Spring, Ashlyn M, Have, Sara Ten, Glascock, Jacqueline J, Gupta, Kushol, Van Duyne, Gregory D, Emanuele, Michael J, Lamond, Angus I, Wagner, Eric J, Lorson, Christian L, Matera, A Gregory
Published in Molecular biology of the cell (15.01.2018)
Published in Molecular biology of the cell (15.01.2018)
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Journal Article
Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCF Slmb degron
Gray, Kelsey M, Kaifer, Kevin A, Baillat, David, Wen, Ying, Bonacci, Thomas R, Ebert, Allison D, Raimer, Amanda C, Spring, Ashlyn M, Have, Sara Ten, Glascock, Jacqueline J, Gupta, Kushol, Van Duyne, Gregory D, Emanuele, Michael J, Lamond, Angus I, Wagner, Eric J, Lorson, Christian L, Matera, A Gregory
Published in Molecular biology of the cell (15.01.2018)
Published in Molecular biology of the cell (15.01.2018)
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Journal Article
AAV9-DOK7 gene therapy reduces disease severity in Smn SMA model mice
Kaifer, Kevin A., Villalón, Eric, Smith, Caley E., Simon, Madeline E., Marquez, Jose, Hopkins, Abigail E., Morcos, Toni I., Lorson, Christian L.
Published in Biochemical and biophysical research communications (10.09.2020)
Published in Biochemical and biophysical research communications (10.09.2020)
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Journal Article
AAV9-DOK7 gene therapy reduces disease severity in Smn 2B/- SMA model mice
Kaifer, Kevin A, Villalón, Eric, Smith, Caley E, Simon, Madeline E, Marquez, Jose, Hopkins, Abigail E, Morcos, Toni I, Lorson, Christian L
Published in Biochemical and biophysical research communications (10.09.2020)
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Published in Biochemical and biophysical research communications (10.09.2020)
Journal Article
Self-oligomerization regulates stability of Survival Motor Neuron (SMN) protein isoforms by sequestering an SCFSlmb degron
Gray, Kelsey M, Kaifer, Kevin A, Baillat, David, Wen, Ying, Bonacci, Thomas R, Ebert, Allison D, Raimer, Amanda C, Spring, Ashlyn M, Sara Ten Have, Glascock, Jacqueline J, Gupta, Kushol, Van Duyne, Gregory D, Emanuele, Michael J, Lamond, Angus I, Wagner, Eric J, Lorson, Christian L, Matera, A Gregory
Published in bioRxiv (06.11.2017)
Published in bioRxiv (06.11.2017)
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