Crystal structure of the pleckstrin homology-phosphotyrosine binding (PH-PTB) targeting region of insulin receptor substrate 1

gan Dhe-Paganon, Sirano, Ottinger, Elizabeth A., Nolte, Robert T., Eck, Michael J., Shoelson, Steven E.
Cyhoeddwyd 1999

Disease models for the development of therapies for lysosomal storage diseases

gan Xu, Miao, Motabar, Omid, Ferrer, Marc, Marugan, Juan J., Zheng, Wei, Ottinger, Elizabeth A.
Cyhoeddwyd yn Ann N Y Acad Sci (2016)

Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses

gan Sima, Ni, Li, Rong, Huang, Wei, Xu, Miao, Beers, Jeanette, Zou, Jizhong, Titus, Steven, Ottinger, Elizabeth A., Marugan, Juan J., Xie, Xing, Zheng, Wei
Cyhoeddwyd yn Orphanet J Rare Dis (2018)

Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease

gan Walkley, Steven U., Davidson, Cristin D., Jacoby, Jonathan, Marella, Philip D., Ottinger, Elizabeth A., Austin, Christopher P., Porter, Forbes D., Vite, Charles H., Ory, Daniel S.
Cyhoeddwyd yn Orphanet J Rare Dis (2016)

Generation of an induced pluripotent stem cell line (TRNDi003-A) from a Noonan syndrome with multiple lentigines (NSML) patient carrying a p.Q510P mutation in the PTPN11 gene

gan Li, Rong, Baskfield, Amanda, Lin, Yongshun, Beers, Jeanette, Zou, Jizhong, Liu, Chengyu, Jaffré, Fabrice, Roberts, Amy E., Ottinger, Elizabeth A., Kontaridis, Maria I., Zheng, Wei
Cyhoeddwyd yn Stem Cell Res (2018)

Phosphocyclocreatine is the dominant form of cyclocreatine in control and creatine transporter deficiency patient fibroblasts

gan Gorshkov, Kirill, Wang, Amy Q., Sun, Wei, Fisher, Ethan, Frigeni, Marta, Singleton, Marc, Thorne, Natasha, Class, Bradley, Huang, Wenwei, Longo, Nicola, Do, Minh‐Ha T., Ottinger, Elizabeth A., Xu, Xin, Zheng, Wei
Cyhoeddwyd yn Pharmacol Res Perspect (2019)

Small Molecule Mimetics of an HIV-1 gp41 Fusion Intermediate as Vaccine Leads

gan Caulfield, Michael J., Dudkin, Vadim Y., Ottinger, Elizabeth A., Getty, Krista L., Zuck, Paul D., Kaufhold, Robin M., Hepler, Robert W., McGaughey, Georgia B., Citron, Michael, Hrin, Renee C., Wang, Ying-Jie, Miller, Michael D., Joyce, Joseph G.
Cyhoeddwyd 2010

The Fungal Cyp51 Inhibitor VT-1129 Is Efficacious in an Experimental Model of Cryptococcal Meningitis

gan Wiederhold, Nathan P., Najvar, Laura K., Garvey, Edward P., Brand, Stephen R., Xu, Xin, Ottinger, Elizabeth A., Alimardanov, Asaf, Cradock, Jim, Behnke, Mark, Hoekstra, William J., Schotzinger, Robert J., Jaramillo, Rosie, Olivo, Marcos, Kirkpatrick, William R., Patterson, Thomas F.
Cyhoeddwyd yn Antimicrob Agents Chemother (2018)

The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

gan Brooks, Philip J., Ottinger, Elizabeth A., Portero, Deanna, Lomash, Richa Madan, Alimardanov, Asaf, Terse, Pramod, Xu, Xin, Chandler, Randy J., Hauserman, Janelle Geist, Esposito, Eric, Bönnemann, Carsten G., Venditti, Charles P., Austin, Christopher P., Pariser, Anne, Lo, Donald C.
Cyhoeddwyd yn Hum Gene Ther (2020)

In Vivo Efficacy of VT-1129 against Experimental Cryptococcal Meningitis with the Use of a Loading Dose-Maintenance Dose Administration Strategy

gan Wiederhold, Nathan P., Xu, Xin, Wang, Amy, Najvar, Laura K., Garvey, Edward P., Ottinger, Elizabeth A., Alimardanov, Asaf, Cradock, Jim, Behnke, Mark, Hoekstra, William J., Brand, Stephen R., Schotzinger, Robert J., Jaramillo, Rosie, Olivo, Marcos, Kirkpatrick, William R., Patterson, Thomas F.
Cyhoeddwyd yn Antimicrob Agents Chemother (2018)

Collaborative Development of 2-Hydroxypropyl-β-Cyclodextrin for the Treatment of Niemann-Pick Type C1 Disease

gan Ottinger, Elizabeth A., Kao, Mark L., Carrillo-Carrasco, Nuria, Yanjanin, Nicole, Shankar, Roopa Kanakatti, Janssen, Marjo, Brewster, Marcus, Scott, Ilona, Xu, Xin, Cradock, Jim, Terse, Pramod, Dehdashti, Seameen, Marugan, Juan, Zheng, Wei, Portilla, Lili, Hubbs, Alan, Pavan, William J., Heiss, John, Vite, Charles H., Walkley, Steven U., Ory, Daniel S., Silber, Steven A., Porter, Forbes D., Austin, Christopher P., McKew, John C.
Cyhoeddwyd 2014

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick Disease, type C1: an ad-hoc analysis of a non-randomized, open-label, phase 1/...

gan Ory, Daniel S., Ottinger, Elizabeth A., Farhat, Nicole Yanjanin, King, Kelly A., Jiang, Xuntian, Weissfeld, Lisa, Berry-Kravis, Elizabeth, Davidson, Cristin D., Bianconi, Simona, Keener, Lee Ann, Rao, Ravichandran, Soldatos, Ariane, Sidhu, Rohini, Walters, Kimberly A., Xu, Xin, Thurm, Audrey, Solomon, Beth, Pavan, William J., Machielse Drs, Bernardus N., Kao, Mark, Silber, Steven A., McKew, John C., Brewer, Carmen C., Vite, Charles H., Walkley, Steven U., Austin, Christopher P., Porter, Forbes D.
Cyhoeddwyd yn Lancet (2017)