未登录 分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence Olaghere, Jimi; Williams, David A.; Farrar, Jeremy; Buening, Hildegard; Calhoun, Cecelia; Ho, Tony; Inamdar, Maneesha S.; Liu, David; Makani, Julie; Nyarko, Kwasi; Ruiz, Sol; Tisdale, John; Mccune, Joseph M.; Boadi, Esther; Reagan Udall Fn FDA 分享 收藏
An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel 关于Lovotibeglogene Autotemcel（Lovo-cel）治疗镰状细胞病（SCD）的临床试验进展及对Lovo-cel反应的早期预测因子分析 Rifkin-Zenenberg, Stacey; Kanter, Julie; Kinney, Melissa A.; Kwiatkowski, Janet L.; Nickel, Robert S.; Walters, Mark C.; Parikh, Suhag; Thompson, Alexis; George, Anil P.; Mapara, Markus; Martin, Paul L.; Chawla, Anjulika; Lodaya, Ankit; Pan, Lin; Sheldon-Waniga, Emily; Pierciey Jr, Francis J.; Tisdale, John; Gupta, Ashish O. 分享 收藏
CD117 Antibody Conditioning and Multiplex Base Editing Enable Rapid and Robust Fetal Hemoglobin Reactivation in a Rhesus Autologous Transplantation Model CD117抗体预处理与多重碱基编辑技术实现灵长类自体移植模型中快速且稳定的胎儿血红蛋白重激活 Demirci, Selami; Mondal, Nandini; Austin, Wayne; Butt, Henna; London, Evan; Sathish, Shruti; Zhang, Kathy; Wong, Jeffrey; Kromer, Hugh; Harmon, Alexander; Decker, Jeremy; Le, Anh; Essawi, Khaled; Krouse, Allen E.; Linde, N. Seth; Bonifacino, Aylin; Engels, Theresa; Golomb, Justin; Hong, So Gun; Uchida, Naoya; Gaudelli, Nicole; Hartigan, Adam J.; Mcdonagh, Charlotte F.; Chu, S. Haihua; Donahue, Robert E.; Tisdale, John 分享 收藏
Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease Lessard, Samuel; Rimmele, Pauline; Ling, Hui; Moran, Kevin; Vieira, Benjamin; Lin, Yi-Dong; Rajani, Gaurav Manohar; Hong, Vu; Reik, Andreas; Boismenu, Richard; Hsu, Ben; Chen, Michael; Cockroft, Bettina M.; Uchida, Naoya; Tisdale, John; Alavi, Asif; Krishnamurti, Lakshmanan; Abedi, Mehrdad; Galeon, Isobelle; Reiner, David; Wang, Lin; Ramezi, Anne; Rendo, Pablo; Walters, Mark C.; Levasseur, Dana; Peters, Robert; Harris, Timothy; Hicks, Alexandra 分享 收藏
Pulmonary Function after Nonmyeloablative Hematopoietic Cell Transplant for Sickle Cell Disease Ruhl, A. Parker; Shalhoub, Ruba; Jeffries, Neal; Limerick, Emily M.; Leonard, Alexis; Barochia, Amisha, V; Tisdale, John F.; Fitzhugh, Courtney D.; Hsieh, Matthew M. 分享 收藏
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+cell therapy to induce fetal hemoglobin for sickle cell disease 新型Cas9基因组编辑的自体CD34细胞疗法诱导胎儿血红蛋白治疗镰状细胞病的开发和IND支持研究 Katta, Varun; O'Keefe, Kiera; Li, Yichao; Mayuranathan, Thiyagaraj; Lazzarotto, Cicera R.; Wood, Rachael K.; Levine, Rachel M.; Powers, Alicia; Mayberry, Kalin; Manquen, Garret; Yao, Yu; Zhang, Jingjing; Jang, Yoonjeong; Nimmagadda, Nikitha; Dempsey, Erin A.; Lee, GaHyun; Uchida, Naoya; Cheng, Yong; Fazio, Frank; Lockey, Tim; Meagher, Mike; Sharma, Akshay; Tisdale, John F.; Zhou, Sheng; Yen, Jonathan S.; Weiss, Mitchell J.; Tsai, Shengdar Q. 分享 收藏
In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation Leonard, Alexis K.; Furstenau, Dana; Inam, Zaina; Luckett, Christina; Chu, Rebecca; Demirci, Selami; Essawi, Khaled; Gudmundsdottir, Bjorg; Hinds, Malikiya; DiNicola, Julia; Li, Quan; Eaton, William A.; Cellmer, Troy; Wang, Xunde; Thein, Swee Lay; Macari, Elizabeth R.; VanNest, Sara; Hsieh, Matthew M.; Bonner, Melissa; Pierciey, Francis J.; Tisdale, John F. 分享 收藏
分享 收藏
分享 收藏
Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model Uchida, Naoya; Stasula, Ulana; Demirci, Selami; Germino-Watnick, Paula; Hinds, Malikiya; Le, Anh; Chu, Rebecca; Berg, Alexander; Liu, Xiong; Su, Ling; Wu, Xiaolin; Krouse, Allen E.; Linde, N. Seth; Bonifacino, Aylin; Hong, So Gun; Dunbar, Cynthia E.; Lanieri, Leanne; Bhat, Anjali; Palchaudhuri, Rahul; Bennet, Bindu; Hoban, Megan; Bertelsen, Kirk; Olson, Lisa M.; Donahue, Robert E.; Tisdale, John F. 分享 收藏
Potent and uniform fetal hemoglobin induction via base editing Mayuranathan, Thiyagaraj; Newby, Gregory A.; Feng, Ruopeng; Yao, Yu; Mayberry, Kalin D.; Lazzarotto, Cicera R.; Li, Yichao; Levine, Rachel M.; Nimmagadda, Nikitha; Dempsey, Erin; Kang, Guolian; Porter, Shaina N.; Doerfler, Phillip A.; Zhang, Jingjing; Jang, Yoonjeong; Chen, Jingjing; Bell, Henry W.; Crossley, Merlin; Bhoopalan, Senthil Velan; Sharma, Akshay; Tisdale, John F.; Pruett-Miller, Shondra M.; Cheng, Yong; Tsai, Shengdar Q.; Liu, David R.; Weiss, Mitchell J.; Yen, Jonathan S. 分享 收藏
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice Everette, Kelcee A.; Newby, Gregory A.; Levine, Rachel M.; Mayberry, Kalin; Jang, Yoonjeong; Mayuranathan, Thiyagaraj; Nimmagadda, Nikitha; Dempsey, Erin; Li, Yichao; Bhoopalan, Senthil Velan; Liu, Xiong; Davis, Jessie R.; Nelson, Andrew T.; Chen, Peter J.; Sousa, Alexander A.; Cheng, Yong; Tisdale, John F.; Weiss, Mitchell J.; Yen, Jonathan S.; Liu, David R. 分享 收藏
Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells 自组织的卵黄囊样类器官允许从诱导的多能干细胞可扩展地生成多能造血祖细胞 Tamaoki, Naritaka; Siebert, Stefan; Maeda, Takuya; Ha, Ngoc-Han; Good, Meghan L.; Huang, Yin; Vodnala, Suman K.; Haro-Mora, Juan J.; Uchida, Naoya; Tisdale, John F.; Sweeney, Colin L.; Choi, Uimook; Brault, Julie; Koontz, Sherry; Malech, Harry L.; Yamazaki, Yasuhiro; Isonaka, Risa; Goldstein, David S.; Kimura, Masaki; Takebe, Takanori; Zou, Jizhong; Stroncek, David F.; Robey, Pamela G.; Kruhlak, Michael J.; Restifo, Nicholas P.; Vizcardo, Raul 分享 收藏
Forced enhancer-promoter rewiring to alter in animal models Peslak, Scott A.; Demirci, Selami; Chandra, Vemika; Ryu, Byoung; Bhardwaj, Saurabh K.; Jiang, Jing; Rupon, Jeremy W.; Throm, Robert E.; Uchida, Naoya; Leonard, Alexis; Essawi, Khaled; Bonifacino, Aylin C.; Krouse, Allen E.; Linde, Nathaniel S.; Donahue, Robert E.; Ferrara, Francesca; Wielgosz, Matthew; Abdulmalik, Osheiza; Hamagami, Nicole; Germino-Watnick, Paula; Le, Anh; Chu, Rebecca; Hinds, Malikiya; Weiss, Mitchell J.; Tong, Wei; Tisdale, John F.; Blobel, Gerd A. 分享 收藏
未登录.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
