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ArticlesPeer ReviewedFull Text
Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG
Author / Creator:Iyer, Sangeetha ; Sam, Feba S ; DiPrimio, Nina ; Preston, Graeme ; Verheijen, Jan ; Murthy, Kausalya ; Parton, Zachary ; Tsang, Hillary ; Lao, Jessica ; Morava, Eva ; Perlstein, Ethan O
Publisher:England: The Company of Biologists Ltd
Journal title:Disease models & mechanisms, 2019-11, Vol.12 (11)
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ArticlesPeer ReviewedFull Text
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
Author / Creator:Asokan, Aravind ; Conway, Julia C ; Phillips, Jana L ; Li, Chengwen ; Hegge, Julia ; Sinnott, Rebecca ; Yadav, Swati ; DiPrimio, Nina ; Nam, Hyun-Joo ; Agbandje-McKenna, Mavis ; McPhee, Scott ; Wolff, Jon ; Samulski, R Jude
Publisher:London: Nature Publishing Group UK
Journal title:Nature biotechnology, 2010-01, Vol.28 (1), p.79-82
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ArticlesPeer ReviewedFull Text
Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles
Author / Creator:Li, Wuping ; Asokan, Aravind ; Wu, Zhijian ; Van Dyke, Terry ; DiPrimio, Nina ; Johnson, Jarrod S ; Govindaswamy, Lakshmanan ; Agbandje-McKenna, Mavis ; Leichtle, Stefan ; Eugene Redmond Jr, D ; McCown, Thomas J ; Petermann, Kimberly B ; Sharpless, Norman E ; Samulski, Richard J
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2008-07, Vol.16 (7), p.1252-1260
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ArticlesPeer ReviewedFull Text
Drug screens of NGLY1 deficiency in worm and fly models reveal catecholamine, NRF2 and anti-inflammatory-pathway activation as potential clinical approaches
Author / Creator:Iyer, Sangeetha ; Mast, Joshua D ; Tsang, Hillary ; Rodriguez, Tamy P ; DiPrimio, Nina ; Prangley, Madeleine ; Sam, Feba S ; Parton, Zachary ; Perlstein, Ethan O
Publisher:England: The Company of Biologists Ltd
Journal title:Disease models & mechanisms, 2019, Vol.12 (11)
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ArticlesPeer ReviewedFull Text
Yeast Models of Phosphomannomutase 2 Deficiency, a Congenital Disorder of Glycosylation
Author / Creator:Lao, Jessica P ; DiPrimio, Nina ; Prangley, Madeleine ; Sam, Feba S ; Mast, Joshua D ; Perlstein, Ethan O
Publisher:Oxford: Oxford University Press
Journal title:G3 : genes - genomes - genetics, 2019-02, Vol.9 (2), p.413-423
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ArticlesPeer ReviewedFull Text
104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle
Author / Creator:DiPrimio, Nina ; Asokan, Aravind ; Samulski, Richard J.
Publisher:Milwaukee: Elsevier Limited
Journal title:Molecular therapy, 2006-05, Vol.13 (S1), p.S43
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ArticlesPeer ReviewedFull Text
Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles
Author / Creator:Li, Wuping ; Asokan, Aravind ; Wu, Zhijian ; Van Dyke, Terry ; DiPrimio, Nina ; Johnson, Jarrod S ; Govindaswamy, Lakshmanan ; Agbandje-McKenna, Mavis ; Leichtle, Stefan ; Eugene Redmond, Jr, D ; McCown, Thomas J ; Petermann, Kimberly B ; Sharpless, Norman E ; Samulski, Richard J
Publisher:United States
Journal title:Molecular therapy, 2008-07, Vol.16 (7), p.1252
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ArticlesFull Text
Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG
Author / Creator:Iyer, Sangeetha ; Sam, Feba S ; Diprimio, Nina ; Preston, Graeme ; Verhejein, Jan ; Murthy, Kausalya ; Parton, Zachary ; Tsang, Hillary ; Lao, Jessica ; Morava, Eva ; Perlstein, Ethan O
Publisher:Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title:bioRxiv, 2019-09
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ArticlesFull Text
Yeast Models Of Phosphomannomutase 2 Deficiency, A Congenital Disorder Of Glycosylation
Author / Creator:Lao, Jessica P ; Diprimio, Nina ; Prangley, Madeleine ; Sam, Feba S ; Mast, Joshua D ; Perlstein, Ethan O
Publisher:Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title:bioRxiv, 2018-09
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ArticlesFull Text
Multi-Species Phenotypic Screening across Disease Models of Mucolipidosis Type IV
Author / Creator:Hadjikyriacou, Andrea ; Iyer, Sangeetha ; Mast, Joshua D ; Diprimio, Nina ; Concannon, John ; Ketterman, Joshua ; Sigoillot, Frederic ; Rodriguez, Tamy P ; Sam, Feba S ; Tsang, Hillary ; Prangley, Madeleine ; Bilen, Julide ; Murthy, Kausalya ; Hartl, Tom A ; Antczak, Christophe ; Jenkins, Jeremy L ; Ross, Nathan T ; Nyfeler, Beat ; Jain, Rishi K ; Tallarico, John A ; Perlstein, Ethan O ; Canham, Stephen M
Publisher:Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title:bioRxiv, 2021-03
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ArticlesFull Text
Drug screens of NGLY1 Deficiency worm and fly models reveal catecholamine, NRF2 and anti-inflammatory pathway activation as potential clinical approaches
Author / Creator:Iyer, Sangeetha ; Mast, Joshua D ; Tsang, Hillary ; Rodriguez, Tamy P ; Diprimio, Nina ; Prangley, Madeleine ; Sam, Feba S ; Parton, Zachary ; Perlstein, Ethan O
Publisher:Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title:bioRxiv, 2019-08
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Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG

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Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

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Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

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Drug screens of NGLY1 deficiency in worm and fly models reveal catecholamine, NRF2 and anti-inflammatory-pathway activation as potential clinical approaches

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Yeast Models of Phosphomannomutase 2 Deficiency, a Congenital Disorder of Glycosylation

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104. Role of the HI Loop in the Adeno- Associated Virus (AAV) Life Cycle

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Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

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Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG

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Yeast Models Of Phosphomannomutase 2 Deficiency, A Congenital Disorder Of Glycosylation

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Multi-Species Phenotypic Screening across Disease Models of Mucolipidosis Type IV

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Drug screens of NGLY1 Deficiency worm and fly models reveal catecholamine, NRF2 and anti-inflammatory pathway activation as potential clinical approaches

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