未登录 Intrathecal Gene Therapy for Giant Axonal Neuropathy Bharucha-Goebel, Diana X.; Todd, Joshua J.; Saade, Dimah; Norato, Gina; Jain, Minal; Lehky, Tanya; Bailey, Rachel M.; Chichester, Jessica A.; Calcedo, Roberto; Armao, Diane; Foley, A. Reghan; Mohassel, Payam; Tesfaye, Eshetu; Carlin, Bradley P.; Seremula, Beth; Waite, Melissa; Zein, Wadih M.; Huryn, Laryssa A.; Crawford, Thomas O.; Sumner, Charlotte J.; Hoke, Ahmet; Heiss, John D.; Charnas, Lawrence; Hooper, Jody E.; Bouldin, Thomas W.; Kang, Elizabeth M.; Rybin, Denis; Gray, Steven J.; Bonnemann, Carsten G. 分享 收藏
An Open Resource for Non-human Primate Optogenetics Tremblay, Sebastien; Acker, Leah; Afraz, Arash; Albaugh, Daniel L.; Amita, Hidetoshi; Andrei, Ariana R.; Angelucci, Alessandra; Aschner, Amir; Balan, Puiu F.; Basso, Michele A.; Benvenuti, Giacomo; Bohlen, Martin O.; Caiola, Michael J.; Calcedo, Roberto; Cavanaugh, James; Chen, Yuzhi; Chen, Spencer; Chernov, Mykyta M.; Clark, Andrew M.; Dai, Ji; Debes, Samantha R.; Deisseroth, Karl; Desimone, Robert; Dragoi, Valentin; Egger, Seth W.; Eldridge, Mark A. G.; El-Nahal, Hala G.; Fabbrini, Francesco; Federer, Frederick; Fetsch, Christopher R.; Fortuna, Michel G.; Friedman, Robert M.; Fujii, Naotaka; Gail, Alexander; Galvan, Adriana; Ghosh, Supriya; Gieselmann, Marc Alwin; Gulli, Roberto A.; Hikosaka, Okihide; Hosseini, Eghbal A.; Hu, Xing; Huer, Janina; Inoue, Ken-ichi; Janz, Roger; Jazayeri, Mehrdad; Jiang, Rundong; Ju, Niansheng; Kar, Kohitij; Klein, Carsten; Kohn, Adam; Komatsu, Misako; Maeda, Kazutaka; Martinez-Trujillo, Julio C.; Matsumoto, Masayuki; Maunsell, John H. R.; Mendoza-Halliday, Diego; Monosov, Ilya E.; Muers, Ross S.; Nurminen, Lauri; Ortiz-Rios, Michael; O'Shea, Daniel J.; Palfi, Stephane; Petkov, Christopher, I; Pojoga, Sorin; Rajalingham, Rishi; Ramakrishnan, Charu; Remington, Evan D.; Revsine, Cambria; Roe, Anna W.; Sabes, Philip N.; Saunders, Richard C.; Scherberger, Hansjorg; Schmid, Michael C.; Schult, Wolfram; Seidemann, Eyal; Senova, Yann-Suhan; Shadlen, Michael N.; Sheinberg, David L.; Siu, Caitlin; Smith, Yoland; Solomon, Selina S.; Sommer, Marc A.; Spudich, John L.; Stauffer, William R.; Takada, Masahiko; Tang, Shiming; Thiele, Alexander; Treue, Stefan; Vanduffel, Wim; Vogels, Rufin; Whitmire, Matthew P.; Wichmann, Thomas; Wurtz, Robert H.; Xu, Haoran; Yazdan-Shahmorad, Azadeh; Shenoy, Krishna, V; DiCarlo, James J.; Pla, Michael L. 分享 收藏
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates Hordeaux, Juliette; Buza, Elizabeth L.; Jeffrey, Brianne; Song, Chunjuan; Jahan, Tahsin; Yuan, Yuan; Zhu, Yanqing; Bell, Peter; Li, Mingyao; Chichester, Jessica A.; Calcedo, Roberto; Wilson, James M. 分享 收藏
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Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol Wang, Lili; Smith, Jeff; Breton, Camilo; Clark, Peter; Zhang, Jia; Ying, Lei; Che, Yan; Lape, Janel; Bell, Peter; Calcedo, Roberto; Buza, Elizabeth L.; Saveliev, Alexei; Bartsevich, Victor V.; He, Zhenning; White, John; Li, Mingyao; Jantz, Derek; Wilson, James M. 分享 收藏
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency Mueller, Christian; Gernoux, Gwladys; Gruntman, Alisha M.; Borel, Florie; Reeves, Emer P.; Calcedo, Roberto; Rouhani, Farshid N.; Yachnis, Anthony; Humphries, Margaret; Campbell-Thompson, Martha; Messina, Louis; Chulay, Jeffrey D.; Trapnell, Bruce; Wilson, James M.; McElvaney, Noel G.; Flotte, Terence R. 分享 收藏
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Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy Weleber, Richard G.; Pennesi, Mark E.; Wilson, David J.; Kaushal, Shalesh; Erker, Laura R.; Jensen, Lauren; McBride, Maureen T.; Flotte, Terence R.; Humphries, Margaret; Calcedo, Roberto; Hauswirth, William W.; Chulay, Jeffrey D.; Stout, J. Timothy 分享 收藏
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Safety and Biodistribution Study of rAAV2tYF-PR1.7-hCNGB3 in CNGB3-Deficient Mice Ye, Guo-jie; Budzynski, Ewa; Sonnentag, Peter; Nork, T. Michael; Miller, Paul E.; McPherson, Leslie; Hoeve, James N. Ver; Smith, Leia M.; Arndt, Tara Arndt; Mandapati, Savitri; Robinson, Paulette M.; Calcedo, Roberto; Knop, David R.; Hauswirth, William W.; Chulay, Jeffrey D. 分享 收藏
Safety and Biodistribution Study of rAAV2tYF-PR1.7-hCNGB3 in Nonhuman Primates Ye, Guo-jie; Budzynski, Ewa; Sonnentag, Peter; Nork, T. Michael; Miller, Paul E.; Sharma, Alok K.; Hoeve, James N. Ver; Smith, Leia M.; Arndt, Tara; Calcedo, Roberto; Gaskin, Chantelle; Robinson, Paulette M.; Knop, David R.; Hauswirth, William W.; Chulay, Jeffrey D. 分享 收藏
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII Gurda, Brittney L.; De Lataillade, Adrien De Guilhem; Bell, Peter; Zhu, Yanqing; Yu, Hongwei; Wang, Ping; Bagel, Jessica; Vite, Charles H.; Sikora, Tracey; Hinderer, Christian; Calcedo, Roberto; Yox, Alexander D.; Steet, Richard A.; Ruane, Therese; O'Donnell, Patricia; Gao, Guangping; Wilson, James M.; Casal, Margret; Ponder, Katherine P.; Haskins, Mark E. 分享 收藏
Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids 基于天然和工程化AAV衣壳的AAV载体肝脏基因转移的比较研究 Wang, Lili; Bell, Peter; Somanathan, Suryanarayan; Wang, Qiang; He, Zhenning; Yu, Hongwei; McMenamin, Deirdre; Goode, Tamara; Calcedo, Roberto; Wilson, James M. 分享 收藏
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial Alton, Eric W. F. W.; Armstrong, David K.; Ashby, Deborah; Bayfield, Katie J.; Bilton, Diana; Bloomfield, Emily V.; Boyd, A. Christopher; Brand, June; Buchan, Ruaridh; Calcedo, Roberto; Carvelli, Paula; Chan, Mario; Cheng, Seng H.; Collie, D. David S.; Cunningham, Steve; Davidson, Heather E.; Davies, Gwyneth; Davies, Jane C.; Davies, Lee A.; Dewar, Maria H.; Doherty, Ann; Donovan, Jackie; Dwyer, Natalie S.; Elgmati, Hala I.; Featherstone, Rosanna F.; Gavino, Jemyr; Gea-Sorli, Sabrina; Geddes, Duncan M.; Gibson, James S. R.; Gill, Deborah R.; Greening, Andrew P.; Griesenbach, Uta; Hansell, David M.; Harman, Katharine; Higgins, Tracy E.; Hodges, Samantha L.; Hyde, Stephen C.; Hyndman, Laura; Innes, J. Alastair; Jacob, Joseph; Jones, Nancy; Keogh, Brian F.; Limberis, Maria P.; Lloyd-Evans, Paul; Maclean, Alan W.; Manvell, Michelle C.; McCormick, Dominique; McGovern, Michael; McLachlan, Gerry; Meng, Cuixiang; Montero, M. Angeles; Milligan, Hazel; Moyce, Laura J.; Murray, Gordon D.; Nicholson, Andrew G.; Osadolor, Tina; Parra-Leiton, Javier; Porteous, David J.; Pringle, Ian A.; Punch, Emma K.; Pytel, Kamila M.; Quittner, Alexandra L.; Rivellini, Gina; Saunders, Clare J.; Scheule, Ronald K.; Sheard, Sarah; Simmonds, Nicholas J.; Smith, Keith; Smith, Stephen N.; Soussi, Najwa; Soussi, Samia; Spearing, Emma J.; Stevenson, Barbara J.; Sumner-Jones, Stephanie G.; Turkkila, Minna; Ureta, Rosa P.; Waller, Michael D.; Wasowicz, Marguerite Y.; Wilson, James M.; Wolstenholme-Hogg, Paul 分享 收藏
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IMMUNE RESPONSES TO SINGLE AND REPEATED ADMINISTRATION OF PGM169/GL67A: THE UK CF GENE THERAPY CONSORTIUM CLINICAL TRIALS Griesenbach, U.; Boyd, A. C.; Calcedo, R.; Cheng, S.; Cunningham, S.; Davies, J. C.; Dewar, M.; Gill, D. R.; Doherty, A.; Higgins, T.; Hyde, S. C.; Manvell, M.; Meng, C.; Innes, J. A.; Limberis, M. P.; Punch, E.; Scheule, R.; Soussi, N.; Soussi, S.; Wilson, J. M.; Alton, Ewfw 分享 收藏
Increased Mucosal CD4+ T Cell Activation in Rhesus Macaques following Vaccination with an Adenoviral Vector Bukh, Irene; Calcedo, Roberto; Roy, Soumitra; Carnathan, Diane G.; Grant, Rebecca; Qin, Qiuyue; Boyd, Surina; Ratcliffe, Sarah J.; Veeder, Christin L.; Bellamy, Scarlett L.; Betts, Michael R.; Wilson, James M. 分享 收藏
Gene therapy for mucopolysaccharidosis VII: evaluation of intrathecal rAAV vectors in the canine model Gurda, Brittney; Wang, Ping; Bell, Peter; Bagel, Jessica; Sikora, Tracey; O'Donnell, Patricia; Zhu, Yanqing; Ruane, Therese; Calcedo, Roberto; Yu, Hongwei; Casal, Margret; Vite, Charles; Ponder, Katherine; Wilson, James; Haskins, Mark 分享 收藏
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression Mueller, Christian; Chulay, Jeffrey D.; Trapnell, Bruce C.; Humphries, Margaret; Carey, Brenna; Sandhaus, Robert A.; McElvaney, Noel G.; Messina, Louis; Tang, Qiushi; Rouhani, Farshid N.; Campbell-Thompson, Martha; Fu, Ann Dongtao; Yachnis, Anthony; Knop, David R.; Ye, Guo-jie; Brantly, Mark; Calcedo, Roberto; Somanathan, Suryanarayan; Richman, Lee P.; Vonderheide, Robert H.; Hulme, Maigan A.; Brusko, Todd M.; Wilson, James M.; Flotte, Terence R. 分享 收藏
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