Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.

Autor: Chalermchai Mitrpant, Paul Porensky, Haiyan Zhou, Loren Price, Francesco Muntoni, Sue Fletcher, Steve D Wilton, Arthur H M Burghes

Publikováno v: PLoS ONE, Vol 8, Iss 4, p e62114 (2013)

Spinal muscular atrophy (SMA) is caused by loss of the Survival Motor Neuron 1 (SMN1) gene, resulting in reduced SMN protein. Humans possess the additional SMN2 gene (or genes) that does produce low level of full length SMN, but cannot adequately com

Externí odkaz: https://doaj.org/article/16a3076a88a84ff4b9853b33f426cf8c

Lumbar Fusion for Active Duty Service Members Performed at an Overseas Military Treatment Facility: A 2-Year Retrospective Analysis

Autor: Daniel S Ikeda, Melissa Meister, Paul Porensky, Hana Yokoi, Vijay M Ravindra

Publikováno v: Military Medicine.

Introduction Among U.S. Military active duty service members, low back pain (LBP) and lumbar radiculopathy are common causes of disability and effect job performance and readiness and can lead to medical separation from the military. Among surgical t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4e09eed89eaf2b4f9dd795f5bcc82b22
https://doi.org/10.1093/milmed/usac193

A large animal model of spinal muscular atrophy and correction of phenotype

Autor: Philipp Odermatt, Arthur H.M. Burghes, Leah Schmelzer, Daniel Schümperli, Stephen J. Kolb, Xiaohui Li, Kathrin Meyer, Brian K. Kaspar, Sandra I Duque, W. David Arnold, Paul Porensky

Publikováno v: Annals of Neurology. 77:399-414

OBJECTIVES Spinal muscular atrophy (SMA) is caused by reduced levels of survival motor neuron (SMN) protein, which results in motoneuron loss. Therapeutic strategies to increase SMN levels including drug compounds, antisense oligonucleotides, and scA

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::9a00d735c26e544280b0c3d901d56aa9
https://doi.org/10.1002/ana.24332

Contributors

Autor: Khalid M. Abbed, Kalil G. Abdullah, Paul D. Ackerman, Yunus Alapan, Vincent J. Alentado, Matthew D. Alvin, Christopher P. Ames, Neel Anand, Paul A. Anderson, Lilyana Angelov, Alireza K. Anissipour, John A. Anson, Ronald I. Apfelbaum, Michael Archdeacon, Paul M. Arnold, Mike W.J. Arun, Harel Arzi, Ahmed J. Awad, Basem I. Awad, Biji Bahuleyan, Mark D. Bain, Lissa C. Baird, Jamie Baisden, Nevan G. Baldwin, Perry A. Ball, Karl E. Balsara, Eli M. Baron, H. Hunt Batjer, Andrew M. Bauer, Thomas W. Bauer, Joshua M. Beckman, Gordon R. Bell, Carlo Bellabarba, E. Emily Bennett, Edward C. Benzel, Darren L. Bergey, Tarun Bhalla, Karin S. Bierbrauer, Mark Bilsky, Harjus Birk, Erica F. Bisson, Christopher Bono, Richard J. Bransford, Darrel S. Brodke, Nathaniel Brooks, Cristian Brotea, Jared R. Brougham, Samuel R. Browd, Robert T. Buckley, Shane Burch, John Butler, Mohamad Bydon, Steven Casha, Jeroen Ceuppens, Andrew K. Chan, Thomas C. Chen, Joseph Cheng, Dean Chou, Tanvir Choudhri, Aaron J. Clark, Adam M. Conley, Paul R. Cooper, Domagoj Coric, Mark Corriveau, Ian P. Côté, Jean-Valery C.E. Coumans, Charles H. Crawford, William T. Curry, Scott D. Daffner, Sedat Dalbayrak, Russell C. DeMicco, Harel Deutsch, Sanjay S. Dhall, Denis J. DiAngelo, Curtis A. Dickman, Shah-Nawaz M. Dodwad, Siena M. Duarte, Zeyd Ebrahim, Gerald W. Eckardt, Bruce L. Ehni, Kurt M. Eichholz, Marc Eichler, Samer K. Elbabaa, Benjamin D. Elder, James B. Elder, Richard G. Ellenbogen, Nancy Epstein, Thomas J. Errico, Yoshua Esquenazi, Daniel K. Fahim, Ehab Farag, Chad W. Farley, Michael G. Fehlings, Frank Feigenbaum, Eoin Fenton, Lisa A. Ferrara, R. David Fessler, Richard G. Fessler, Michael A. Finn, Ryan Finnan, Jeffrey S. Fischgrund, Kevin T. Foley, Ricardo B.V. Fontes, Todd B. Francis, Brett A. Freedman, Frederick Frost, John George, John W. German, Peter C. Gerszten, George M. Ghobrial, Zoher Ghogawala, Justin L. Gibson, Christopher C. Gillis, Vijay K. Goel, Jan Goffin, Ziya L. Gokaslan, Sohrab Gollogly, C. Rory Goodwin, Carlos R. Goulart, Vadim Goz, Yair M. Gozal, Randall B. Graham, Gerald A. Grant, Jian Guan, Ilker Gulec, Yazeed M. Gussous, Richard D. Guyer, David Gwinn, Sung Ha, Eldad Hadar, Clayton L. Haldeman, Alexander Y. Halim, Kimberly M. Hamilton, Christine L. Hammer, Fadi Hanbali, Shannon W. Hann, Jurgen Harms, James S. Harrop, Blaine L. Hart, David J. Hart, Daniel Harwell, Reyaad A. Hayek, Robert F. Heary, Fraser C. Henderson, Patrick W. Hitchon, Daniel J. Hoh, Paul J. Holman, Noboru Hosono, Clifford Houseman, John K. Houten, Joseph C. Hsieh, Wellington K. Hsu, Meng Huang, R. John Hurlbert, Lee Hwang, Steven Hwang, Serkan İnceoğlu, Libby Kosnik Infinger, Tatiana von Hertwig Fernandes de Oliveira, Devesh Jalan, Neilank Jha, J. Patrick Johnson, Charles I. Jones, G. Alexander Jones, Michael Jones, Rupa G. Juthani, Christopher D. Kager, Maziyar A. Kalani, M. Yashar S. Kalani, Iain H. Kalfas, Ricky R. Kalra, Reza J. Karimi, Osama Kashlan, Manish K. Kasliwal, Vikas Kaul, Mayank Kaushal, Tyler J. Kenning, Saad Khairi, Tagreed Khalaf, Jad G. Khalil, Larry T. Khoo, Ali Kiapour, Daniel H. Kim, David H. Kim, Kristopher T. Kimmell, Steven Kirshblum, Sameer A. Kitab, Paul Klimo, Eric O. Klineberg, Tyler R. Koski, Thomas A. Kosztowski, Robert J. Kowalski, Ajit A. Krishnaney, Kelly Krupa, Kristin Krupa, Varun R. Kshettry, Sunil Kukreja, Charles Kuntz, Shekar N. Kurpad, Srinivasu Kusuma, Michael LaBagnara, Frank La Marca, Ilya Laufer, Elizabeth Demers Lavelle, William F. Lavelle, W. Thomas Lawrence, Darren R. Lebl, Bryan S. Lee, Sun-Ho Lee, Lawrence G. Lenke, Steven P. Leon, Amy Li, Yiping Li, Isador H. Lieberman, James K.C. Liu, Victor P. Lo, S. Scott Lollis, Miguel Lopez-Gonzalez, Daniel Lubelski, Mark G. Luciano, Andre G. Machado, Raghu Maddela, Ravichandra A. Madineni, Casey Madura, Dennis J. Maiman, David G. Malone, Antonios Mammis, Satyajit Marawar, Nicolas Marcotte, Joseph C. Maroon, Michael D. Martin, Eduardo Martinez-del-Campo, Eric M. Massicotte, Tobias A. Mattei, Paul K. Maurer, Eric A.K. Mayer, Miguel Mayol del Valle, Daniel J. Mazanec, Paul C. McCormick, William McCormick, Zachary A. Medress, Ehud Mendel, Umesh S. Metkar, Vincent J. Miele, Ahmed Mohyeldin, Jad Bou Monsef, Timothy A. Moore, Hikaru Morisue, Peter Morone, Thomas E. Mroz, Jeffrey P. Mullin, F. Reed Murtagh, Ryan D. Murtagh, Sait Naderi, Usha D. Nagaraj, Charles C. Nalley, Anil Nanda, Richard J. Nasca, Anick Nater, Matthew T. Neal, Russ P. Nockels, John A. Norwig, Solomon M. Ondoma, Akinwunmi Oni-Orisan, Jonathan H. Oren, Jennifer Orning, R. Douglas Orr, Katie Orrico, Joseph A. Osorio, Ernesto Otero-Lopez, John O'Toole, Paul Park, Vikas Parmar, Robert S. Pashman, Rakesh D. Patel, Smruti K. Patel, Mick J. Perez-Cruet, Noel I. Perin, David B. Pettigrew, H. Westley Phillips, Rick Placide, Paul Porensky, Joshua P. Prager, Srinivas Prasad, Mark L. Prasarn, Rakesh Ramakrishnan, Ashwin G. Ramayya, Y. Raja Rampersaud, Peter A. Rasmussen, John K. Ratliff, Wolfgang Rauschning, Glenn R. Rechtine, Pablo F. Recinos, Daniel K. Resnick, Jay Rhee, Laurence D. Rhines, Alexander R. Riccio, Marlin Dustin Richardson, Bertram Richter, Ron Riesenburger, K. Daniel Riew, Matthew Rogers, Fanor M. Saavedra, Mina G. Safain, Rajiv Saigal, Paul D. Sawin, Justin K. Scheer, Joshua Scheidler, David W. Schippert, Richard Schlenk, Bradley Schmidt, Meic H. Schmidt, Daniel M. Sciubba, Christopher I. Shaffrey, Mark E. Shaffrey, Anoli Shah, Alok Sharan, Ashwini D. Sharan, Daniel Shedid, Steven Shook, Michael P. Silverstein, Venita M. Simpson, Anthony Sin, Harminder Singh, Donald A. Smith, Gabriel A. Smith, Justin S. Smith, Kyle A. Smith, Volker K.H. Sonntag, Hector Soriano-Baron, Robert F. Spetzler, W. Ryan Spiker, Blake Staub, Michael P. Steinmetz, Charles B. Stillerman, Andrea Strayer, Gandhivarma Subramaniam, Hamdi G. Sukkarieh, Andrew Sumich, Derrick Y. Sun, Tarek P. Sunna, Durga R. Sure, Richard A. Tallarico, Lee A. Tan, Claudio E. Tatsui, Fernando Techy, Nicholas Theodore, Alexander A. Theologis, Nicholas W.M. Thomas, Brian D. Thorp, Scott Tintle, Stavropoula Tjoumakaris, William D. Tobler, Daisuke Togawa, David Traul, Vincent C. Traynelis, A. Sophia Tritle, Gregory R. Trost, Eve C. Tsai, Kene Ugokwe, Kutlauy Uluc, Juan S. Uribe, Alexander R. Vaccaro, Alex Valadka, Aditya Vedantam, Anand Veeravagu, Kushagra Verma, Todd Vitaz, Jean-Marc Voyadzis, Scott Wagner, Trevor C. Wahlquist, Robert Waldrop, Kevin M. Walsh, Jeffrey C. Wang, Michael Y. Wang, Patrick T. Wang, John D. Ward, Zabi Wardak, Connor Wathen, Philip R. Weinstein, Michael Weisman, William C. Welch, Simcha J. Weller, L. Erik Westerlund, Jonathan A. White, Robert G. Whitmore, Jack E. Wilberger, Kim A. Williams, Ethan A. Winkler, Christopher D. Witiw, Christopher E. Wolfla, Jean-Paul Wolinsky, Cyrus Wong, Eric J. Woodard, Vijay Yanamadala, Daniel S. Yanni, Philip A. Yazbak, Chun-Po Yen, Mesut Yilmaz, Narayan Yoganandan, Kenneth S. Yonemura, Kazuo Yonenobu, Hansen A. Yuan, John K. Yue, Adam M. Zanation, Salvatore M. Zavarella, Seth M. Zeidman, Mehmet Zileli, Scott Zuckerman, Holly Zywicke

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::09b577421df1b70fc033d6759bc8d7e5
https://doi.org/10.1016/b978-0-323-40030-5.00219-7

Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy

Autor: Arthur H.M. Burghes, Paul Porensky

Publikováno v: Human Gene Therapy. 24:489-498

Spinal muscular atrophy (SMA) is an autosomal recessive disease affecting ∼1 in 10,000 live births. The most striking component is the loss of α-motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventuall

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2f85f8ffa626c3caaa5436b060816d2a
https://doi.org/10.1089/hum.2012.225