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

Precise targeting of HIV broadly neutralizing antibody precursors in humans

精准靶向人类体内HIV广谱中和抗体前体

Tom G Caniels #,Madhu Prabhakaran #,Gabriel Ozorowski,Kellie J MacPhee,Weiwei Wu,Karlijn van der Straten,Sashank Agrawal,Ronald Derking,Emma I M M Reiss,Katrina Millard,Martina Turroja,Aimee Desrosiers,Jeffrey Bethony,Elissa Malkin,Marinus H Liesdek,Annelou van der Veen,Michelle Klouwens,Jonne L Snitselaar,Joey H Bouhuijs,Rhianna Bronson,Jalen Jean-Baptiste,Suprabhath Gajjala,Zahra Rikhtegaran Tehrani,Alison Benner,Mukundhan Ramaswami,Michael O Duff,Yung-Wen Liu,Alicia H Sato,Ju Yeong Kim,Isabel J L Baken,Catarina Mendes Silva,Tom P L Bijl,Jacqueline van Rijswijk,Judith A Burger,Albert Cupo,Anila Yasmeen,Swastik Phulera,Wen-Hsin Lee,Kipchoge N Randall Jr,Shiyu Zhang,Martin M Corcoran,Isabel Regadas,Alex C Sullivan,David M Brown,Jennifer A Bohl,Kelli M Greene,Hongmei Gao,Nicole L Yates,Sheetal Sawant,Jan M Prins,Neeltje A Kootstra,Stephen M Kaminsky,Burc Barin,Farhad Rahaman,Margaret Meller,Vince Philiponis,Dagna S Laufer,Angela Lombardo,Lindsey Mwoga,Solmaz Shotorbani,Drienna Holman,Richard A Koup,Per Johan Klasse,Gunilla B Karlsson Hedestam,Georgia D Tomaras,Marit J van Gils,David C Montefiori,Adrian B McDermott,Ollivier Hyrien,John P Moore,Ian A Wilson,Andrew B Ward,David J Diemert,Godelieve J de Bree,Sarah F Andrews,Marina Caskey,Rogier W Sanders

Expression and processing of mature human frataxin after gene therapy in mice

小鼠基因治疗后成熟人类 frataxin 的表达和加工

Teerapat Rojsajjakul, Nithya Selvan, Bishnu De, Jonathan B Rosenberg, Stephen M Kaminsky, Dolan Sondhi, Peter Janki, Ronald G Crystal, Clementina Mesaros, Richie Khanna, Ian A Blair

Expression and processing of mature human frataxin after gene therapy in mice

小鼠基因治疗后成熟人类 frataxin 的表达和加工

Teerapat Rojsajjakul, Nithya Selvan, Bishnu De, Jonathan B Rosenberg, Stephen M Kaminsky, Dolan Sondhi, Peter Janki, Ronald G Crystal, Clementina Mesaros, Richie Khanna, Ian A Blair

Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2

通过直接向脑实质注射表达 CLN2 的 rh.10 腺相关病毒来减缓晚期婴儿巴顿病

Dolan Sondhi, Stephen M Kaminsky, Neil R Hackett, Odelya E Pagovich, Jonathan B Rosenberg, Bishnu P De, Alvin Chen, Benjamin Van de Graaf, Jason G Mezey, Grace W Mammen, Denesy Mancenido, Fang Xu, Barry Kosofsky, Kaleb Yohay, Stefan Worgall, Robert J Kaner, Mark Souwedaine, Bruce M Greenwald, Michae

Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin

利用抗氧化的人类 α1-抗胰蛋白酶对 α1-抗胰蛋白酶缺乏症进行基因治疗

Meredith L Sosulski, Katie M Stiles, Esther Z Frenk, Fiona M Hart, Yuki Matsumura, Bishnu P De, Stephen M Kaminsky, Ronald G Crystal

Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema

遗传性血管性水肿小鼠模型中 C1 酯酶抑制剂缺乏症的基因治疗

Ting Qiu, Maria J Chiuchiolo, Adele S Whaley, Anthony R Russo, Dolan Sondhi, Stephen M Kaminsky, Ronald G Crystal, Odelya E Pagovich

Accurate Quantification and Characterization of Adeno-Associated Viral Vectors

腺相关病毒载体的准确定量和表征

David Dobnik, Polona Kogovšek, Tjaša Jakomin, Nejc Košir, Magda Tušek Žnidarič, Maja Leskovec, Stephen M Kaminsky, Janet Mostrom, Hyunmi Lee, Maja Ravnikar

Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease)

脑脊液非靶向代谢物分析揭示晚期婴儿神经元蜡样脂褐素沉积症(CLN2,巴顿病)严重程度的生物标志物

Miriam Sindelar, Jonathan P Dyke, Ruba S Deeb, Dolan Sondhi, Stephen M Kaminsky, Barry E Kosofsky, Douglas J Ballon, Ronald G Crystal, Steven S Gross

AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

AAVrh.10 介导的 APOE2 中枢神经系统基因疗法治疗 APOE4 相关阿尔茨海默病

Jonathan B Rosenberg, Michael G Kaplitt, Bishnu P De, Alvin Chen, Thomas Flagiello, Christiana Salami, Eduard Pey, Lingzhi Zhao, Rodolfo J Ricart Arbona, Sebastien Monette, Jonathan P Dyke, Douglas J Ballon, Stephen M Kaminsky, Dolan Sondhi, Gregory A Petsko, Steven M Paul, Ronald G Crystal

Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice

抗 Tau 单克隆抗体 PHF1 的载体脑内免疫显著降低了突变 Tau 转基因小鼠的 Tau 病理

Wencheng Liu, Lingzhi Zhao, Brittany Blackman, Mayur Parmar, Man Ying Wong, Thomas Woo, Fangmin Yu, Maria J Chiuchiolo, Dolan Sondhi, Stephen M Kaminsky, Ronald G Crystal, Steven M Paul