Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
Coelho, Teresa, Adams, David, Silva, Ana, Lozeron, Pierre, Hawkins, Philip N, Mant, Timothy, Perez, Javier, Chiesa, Joseph, Warrington, Steve, Tranter, Elizabeth, Munisamy, Malathy, Falzone, Rick, Harrop, Jamie, Cehelsky, Jeffrey, Bettencourt, Brian R, Geissler, Mary, Butler, James S, Sehgal, Alfica, Meyers, Rachel E, Chen, Qingmin, Borland, Todd, Hutabarat, Renta M, Clausen, Valerie A, Alvarez, Rene, Fitzgerald, Kevin, Gamba-Vitalo, Christina, Nochur, Saraswathy V, Vaishnaw, Akshay K, Sah, Dinah W.Y, Gollob, Jared A, Suhr, Ole B
Published in The New England journal of medicine (29.08.2013)
Published in The New England journal of medicine (29.08.2013)
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Journal Article
Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis
Butler, James S., Chan, Amy, Costelha, Susete, Fishman, Shannon, Willoughby, Jennifer L. S., Borland, Todd D., Milstein, Stuart, Foster, Donald J., Gonçalves, Paula, Chen, Qingmin, Qin, June, Bettencourt, Brian R., Sah, Dinah W., Alvarez, Rene, Rajeev, Kallanthottathil G., Manoharan, Muthiah, Fitzgerald, Kevin, Meyers, Rachel E., Nochur, Saraswathy V., Saraiva, Maria J., Zimmermann, Tracy S.
Published in Amyloid (01.06.2016)
Published in Amyloid (01.06.2016)
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Journal Article
Dynamics of equilibrium structural fluctuations of apomyoglobin measured by fluorescence correlation spectroscopy
Chen, Huimin, Rhoades, Elizabeth, Butler, James S, Loh, Stewart N, Webb, Watt W
Published in Proceedings of the National Academy of Sciences - PNAS (19.06.2007)
Published in Proceedings of the National Academy of Sciences - PNAS (19.06.2007)
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A competitive enzyme-linked immunosorbent assay specific for murine hepcidin-1: correlation with hepatic mRNA expression in established and novel models of dysregulated iron homeostasis
Gutschow, Patrick, Schmidt, Paul J, Han, Huiling, Ostland, Vaughn, Bartnikas, Thomas B, Pettiglio, Michael A, Herrera, Carolina, Butler, James S, Nemeth, Elizabeta, Ganz, Tomas, Fleming, Mark D, Westerman, Mark
Published in Haematologica (Roma) (01.02.2015)
Published in Haematologica (Roma) (01.02.2015)
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Journal Article
Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms
Akinc, Akin, Querbes, William, De, Soma, Qin, June, Frank-Kamenetsky, Maria, Jayaprakash, K. Narayanannair, Jayaraman, Muthusamy, Rajeev, Kallanthottathil G, Cantley, William L, Dorkin, J Robert, Butler, James S, Qin, LiuLiang, Racie, Timothy, Sprague, Andrew, Fava, Eugenio, Zeigerer, Anja, Hope, Michael J, Zerial, Marino, Sah, Dinah WY, Fitzgerald, Kevin, Tracy, Mark A, Manoharan, Muthiah, Koteliansky, Victor, Fougerolles, Antonin de, Maier, Martin A
Published in Molecular therapy (01.07.2010)
Published in Molecular therapy (01.07.2010)
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CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
Longhurst, Hilary J., Lindsay, Karen, Petersen, Remy S., Fijen, Lauré M., Gurugama, Padmalal, Maag, David, Butler, James S., Shah, Mrinal Y., Golden, Adele, Xu, Yuanxin, Boiselle, Carri, Vogel, Joseph D., Abdelhady, Ahmed M., Maitland, Michael L., McKee, Mark D., Seitzer, Jessica, Han, Bo W., Soukamneuth, Samantha, Leonard, John, Sepp-Lorenzino, Laura, Clark, Eliana D., Lebwohl, David, Cohn, Danny M.
Published in The New England journal of medicine (01.02.2024)
Published in The New England journal of medicine (01.02.2024)
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Journal Article
Combination therapy with a Tmprss6 RNAi‐therapeutic and the oral iron chelator deferiprone additively diminishes secondary iron overload in a mouse model of β‐thalassemia intermedia
Schmidt, Paul J., Racie, Tim, Westerman, Mark, Fitzgerald, Kevin, Butler, James S., Fleming, Mark D.
Published in American journal of hematology (01.04.2015)
Published in American journal of hematology (01.04.2015)
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Journal Article
Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression
Willoughby, Jennifer L.S., Chan, Amy, Sehgal, Alfica, Butler, James S., Nair, Jayaprakash K., Racie, Tim, Shulga-Morskaya, Svetlana, Nguyen, Tuyen, Qian, Kun, Yucius, Kristina, Charisse, Klaus, van Berkel, Theo J.C., Manoharan, Muthiah, Rajeev, Kallanthottathil G., Maier, Martin A., Jadhav, Vasant, Zimmermann, Tracy S.
Published in Molecular therapy (03.01.2018)
Published in Molecular therapy (03.01.2018)
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Journal Article
Bacteriophage P22 Tail Accessory Factor GP26 Is a Long Triple-stranded Coiled-coil
Andrews, Dewan, Butler, James S., Al-Bassam, Jawdat, Joss, Lisa, Winn-Stapley, Danella A., Casjens, Sherwood, Cingolani, Gino
Published in The Journal of biological chemistry (18.02.2005)
Published in The Journal of biological chemistry (18.02.2005)
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RNAi‐mediated reduction of hepatic Tmprss6 diminishes anemia and secondary iron overload in a splenectomized mouse model of β‐thalassemia intermedia
Schmidt, Paul J., Liu, Kaifeng, Visner, Gary, Fitzgerald, Kevin, Fishman, Shannon, Racie, Tim, Hettinger, Julia L., Butler, James S., Fleming, Mark D.
Published in American journal of hematology (01.06.2018)
Published in American journal of hematology (01.06.2018)
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Journal Article
Harnessing a physiologic mechanism for siRNA delivery with mimetic lipoprotein particles
Nakayama, Tomoko, Butler, James S, Sehgal, Alfica, Severgnini, Mariano, Racie, Tim, Sharman, Jennifer, Ding, Feng, Morskaya, Svetlana Shulga, Brodsky, Joshua, Tchangov, Lubomir, Kosovrasti, Verbena, Meys, Mike, Nechev, Lubomir, Wang, Gang, Peng, Chang Geng, Fang, Yupang, Maier, Martin, Rajeev, Kallanthottathil G, Li, Robert, Hettinger, Julia, Barros, Scott, Clausen, Valerie, Zhang, Xuemei, Wang, Qianfan, Hutabarat, Renta, Dokholyan, Nikolay V, Wolfrum, Christian, Manoharan, Muthiah, Kotelianski, Victor, Stoffel, Markus, Sah, Dinah Wy
Published in Molecular therapy (01.08.2012)
Published in Molecular therapy (01.08.2012)
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Journal Article
Clinical outcomes in extracranial tumor sites and unusual toxicities with concurrent whole brain radiation (WBRT) and Erlotinib treatment in patients with non-small cell lung cancer (NSCLC) with brain metastasis
Olmez, Inan, Donahue, Bernadine R, Butler, James S, Huang, Yiwu, Rubin, Philip, Xu, Yiqing
Published in Lung cancer (Amsterdam, Netherlands) (01.11.2010)
Published in Lung cancer (Amsterdam, Netherlands) (01.11.2010)
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Journal Article
850c RNAi Therapeutics Ameliorate Liver Disease Associated with Alpha-1-Antitrypsin Deficiency
Sehgal, Alfica, Blomenkamp, Keith S, Qian, Kun, Fishman, Shannon, Butler, James S, Milstein, Stuart, Kuchimanch, Satya, Barros, Scott, Bettencourt, Brian, Charisse, Klaus B, Simon, Amy, Bumcrot, David, Fitzgerald, Kevin, Teckman, Jeffrey
Published in Gastroenterology (New York, N.Y. 1943) (01.05.2014)
Published in Gastroenterology (New York, N.Y. 1943) (01.05.2014)
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