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

Monitoring biological effects of somatic cell genome editing

监测体细胞基因组编辑的生物学效应

Freedman, Benjamin S; Bulte, Jeff W M; Conklin, Bruce R; Judge, Luke M; Dwinell, Melinda R; Geurts, Aron M; Sitton, Madeleine J; Mahajan, Vineet; Kiani, Samira; Gersbach, Charles A; Ebrahimkhani, Mo R; Kelly, John J; Ronald, John A; Morizane, Ryuji; Gupta, Navin; Shakeri-Zadeh, Ali; Vo, Nicole; Saha, Krishanu; Saxena, Shivani; Gamm, David M; Sinha, Divya; Tarantal, Alice F; Vandsburger, Moriel; Matsubara, Azusa; Fu, Hongxia; Tsai, Shengdar Q

A porcine commotio retinae model for preclinical evaluation of posttraumatic photoreceptor degeneration.

猪视网膜震荡模型用于创伤后感光细胞变性的临床前评估。

Amaral Juan, Bunea Irina, Maminishkis Arvydas, Campos Maria M, Barone Francesca, Gupta Rohan, Farnoodian Mitra, Newport Jonathan, Phillips M Joseph, Sharma Ruchi, Gamm David M, Bharti Kapil, Blanch Richard J

Metabolic stress and early cell death in photoreceptor precursor cells following retinal transplantation

视网膜移植后感光细胞前体细胞的代谢应激和早期细胞死亡

Sudharsan, Raghavi; Dolgova, Natalia; Kwok, Jennifer; Gray, Alexa; Sato, Yu; Madrigal, Agustin Luz; Susaimanickam, Praveen Joseph; Kriukov, Emil; Baranov, Petr; Wolfe, John H; Aguirre, Gustavo D; Gamm, David M; Beltran, William A

Robust generation of photoreceptor-dominant retinal organoids from porcine induced pluripotent stem cells

利用猪诱导多能干细胞高效生成以感光细胞为主的视网膜类器官

Edwards, Kimberly L; Moore, Bethany M; Ganser, Tyler-Serie; Susaimanickam, Praveen Joseph; Sovell, Kai; Martin, Yolana; Jager, Lindsey D; Willes, Ashley M; Moyer, Tyra H; Bowar, Lydia; Phillips, M Joseph; Stewart, Ron; Chu, Li-Fang; Gamm, David M

Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines.

在小鼠和猪动物模型中对视网膜下注射的 Cas9 核糖核蛋白进行评估,突显了基因药物治疗转化中的关键考虑因素

Wei Spencer C, Cantor Aaron J, Walleshauser Jack, Mepani Rina, Melton Kory, Bans Ashil, Khekare Prachi, Gupta Suhani, Wang Jonathan, Soares Craig, Kiwan Radwan, Lee Jieun, McCawley Shannon, Jani Vihasi, Leong Weng In, Shahi Pawan K, Chan Jean, Boivin Pierre, Otoupal Peter, Pattnaik Bikash R, Gamm David M, Saha Krishanu, Gowen Benjamin G, Haak-Frendscho Mary, Janatpour Mary J, Silverman Adam P

Preventing vision loss in a mouse model of Leber Congenital Amaurosis by engineered tRNA

利用基因工程改造的tRNA预防Leber先天性黑蒙小鼠模型的视力丧失

Akyuz, Enes; Shahi, Pawan K; Gissot, Lionel; Saneh, Ahmad Al; Sinha, Divya; Hanstad, Giovanni M; Kabra, Meha; Zepeda, Maria A Fernandez; Gamm, David M; Young, Samuel M; Ahern, Christopher A; Pattnaik, Bikash R

Genome-Wide CRISPR Screening Identifies Cellular Factors Controlling Nonviral Genome Editing Efficiency

全基因组 CRISPR 筛选鉴定出控制非病毒基因组编辑效率的细胞因子

Saxena, Shivani; Kabra, Meha; Abdeen, Amr; Sinha, Divya; Zhu, Min; Xie, Ruosen; Hanstad, Giovanni; Zepeda, Maria A Fernandez; Gamm, David M; Pattnaik, Bikash R; Gong, Shaoqin; Saha, Krishanu

Cone photoreceptors in human stem cell-derived retinal organoids demonstrate intrinsic light responses that mimic those of primate fovea

人类干细胞衍生的视网膜类器官中的锥状感光细胞表现出与灵长类动物中央凹相似的内在光反应。

Saha, Aindrila; Capowski, Elizabeth; Fernandez Zepeda, Maria A; Nelson, Emma C; Gamm, David M; Sinha, Raunak

Systemic immunosuppression promotes survival and integration of subretinally implanted human ESC-derived photoreceptor precursors in dogs

全身免疫抑制可促进犬视网膜下植入的人类胚胎干细胞衍生的光感受器前体细胞的存活和整合。

Ripolles-Garcia, Ana; Dolgova, Natalia; Phillips, M Joseph; Savina, Svetlana; Ludwig, Allison L; Stuedemann, Sara A; Nlebedum, Uchenna; Wolfe, John H; Garden, Oliver A; Maminishkis, Arvydas; Amaral, Juan; Bharti, Kapil; Gamm, David M; Aguirre, Gustavo D; Beltran, William A

The NIH Somatic Cell Genome Editing program

美国国立卫生研究院体细胞基因组编辑计划

Saha, Krishanu; Sontheimer, Erik J; Brooks, P J; Dwinell, Melinda R; Gersbach, Charles A; Liu, David R; Murray, Stephen A; Tsai, Shengdar Q; Wilson, Ross C; Anderson, Daniel G; Asokan, Aravind; Banfield, Jillian F; Bankiewicz, Krystof S; Bao, Gang; Bulte, Jeff W M; Bursac, Nenad; Campbell, Jarryd M; Carlson, Daniel F; Chaikof, Elliot L; Chen, Zheng-Yi; Cheng, R Holland; Clark, Karl J; Curiel, David T; Dahlman, James E; Deverman, Benjamin E; Dickinson, Mary E; Doudna, Jennifer A; Ekker, Stephen C; Emborg, Marina E; Feng, Guoping; Freedman, Benjamin S; Gamm, David M; Gao, Guangping; Ghiran, Ionita C; Glazer, Peter M; Gong, Shaoqin; Heaney, Jason D; Hennebold, Jon D; Hinson, John T; Khvorova, Anastasia; Kiani, Samira; Lagor, William R; Lam, Kit S; Leong, Kam W; Levine, Jon E; Lewis, Jennifer A; Lutz, Cathleen M; Ly, Danith H; Maragh, Samantha; McCray, Paul B Jr; McDevitt, Todd C; Mirochnitchenko, Oleg; Morizane, Ryuji; Murthy, Niren; Prather, Randall S; Ronald, John A; Roy, Subhojit; Roy, Sushmita; Sabbisetti, Venkata; Saltzman, W Mark; Santangelo, Philip J; Segal, David J; Shimoyama, Mary; Skala, Melissa C; Tarantal, Alice F; Tilton, John C; Truskey, George A; Vandsburger, Moriel; Watts, Jonathan K; Wells, Kevin D; Wolfe, Scot A; Xu, Qiaobing; Xue, Wen; Yi, Guohua; Zhou, Jiangbing