日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Intrathecal Gene Therapy for Giant Axonal Neuropathy

鞘内基因疗法治疗巨轴突神经病

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; Bönnemann, Carsten G

An Open Resource for Non-human Primate Optogenetics

非人灵长类动物光遗传学开放资源

Tremblay, Sébastien; 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, Michal 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; Hüer, 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, Stéphane; Petkov, Christopher I; Pojoga, Sorin; Rajalingham, Rishi; Ramakrishnan, Charu; Remington, Evan D; Revsine, Cambria; Roe, Anna W; Sabes, Philip N; Saunders, Richard C; Scherberger, Hansjörg; Schmid, Michael C; Schultz, 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; Platt, Michael L

Susceptibility to SIV Infection After Adenoviral Vaccination in a Low Dose Rhesus Macaque Challenge Model

低剂量恒河猴攻毒模型中腺病毒疫苗接种后对SIV感染的易感性

Brody, Irene Bukh; Calcedo, Roberto; Connell, Mary J; Carnathan, Diane G; Nason, Martha; Lawson, Benton O; Nega, Melon T; Boyd, Surina; Qin, Qiuyue; Vanderford, Thomas H; Wilson, Jolaine M; Wilson, James M; Silvestri, Guido; Betts, Michael R

Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques

优化腺相关病毒介导的人类凝血因子VIII基因治疗在食蟹猴中的应用

Greig, Jenny A; Nordin, Jayme M L; White, John W; Wang, Qiang; Bote, Erin; Goode, Tamara; Calcedo, Roberto; Wadsworth, Samuel; Wang, Lili; Wilson, James M

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

α1-抗胰蛋白酶缺乏症基因治疗后5年表达和中性粒细胞缺陷修复

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

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

在α-1-抗胰蛋白酶缺乏症的基因治疗临床试验中,针对多态性肽的I类限制性T细胞反应

Calcedo, Roberto; Somanathan, Suryanarayan; Qin, Qiuyue; Betts, Michael R; Rech, Andrew J; Vonderheide, Robert H; Mueller, Christian; Flotte, Terence R; Wilson, James M

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR(+/-) Rhesus Macaques

非临床研究:检测AAV8.TBG.hLDLR载体对饲喂普通饲料的野生型和LDLR(+/-)恒河猴的毒性

Greig, Jenny A; Limberis, Maria P; Bell, Peter; Chen, Shu-Jen; Calcedo, Roberto; Rader, Daniel J; Wilson, James M

Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs

表达人或犬 CNGB3 的 AAV5 载体在 CNGB3 突变犬中的安全性和有效性

Ye, Guo-Jie; Komáromy, András M; Zeiss, Caroline; Calcedo, Roberto; Harman, Christine D; Koehl, Kristin L; Stewart, Gabriel A; Iwabe, Simone; Chiodo, Vince A; Hauswirth, William W; Aguirre, Gustavo D; Chulay, Jeffrey D

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia

在纯合子家族性高胆固醇血症小鼠模型中,AAV8.TBG.mLDLR 和 AAV8.TBG.hLDLR 的非临床药理学/毒理学研究

Greig, Jenny A; Limberis, Maria P; Bell, Peter; Chen, Shu-Jen; Calcedo, Roberto; Rader, Daniel J; Wilson, James M

Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia

患有 mut 甲基丙二酸血症的患者体内针对腺相关病毒衣壳的中和抗体

Harrington, Elizabeth A; Sloan, Jennifer L; Manoli, Irini; Chandler, Randy J; Schneider, Mark; McGuire, Peter J; Calcedo, Roberto; Wilson, James M; Venditti, Charles P