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

A novel modifier gene therapy to treat Stargardt disease: Phase 1 GARDian1 Trial Insights

一种治疗斯塔加特病的新型修饰基因疗法:GARDian1 一期试验结果

Khanani, Arshad M; Vajzovic, Lejla; Bakall, Benjamin; Chavali, Venkata Ramana Murthy; Qamar, Huma

Variants in CFAP410 cause a range of retinal and skeletal phenotypes

CFAP410基因变异会导致一系列视网膜和骨骼表型。

Schmidt, Ryan E; Pohodich, Amy E; Birch, David; Jones, Kaylie; Lam, Byron L; Jung, Emily H; Jain, Nieraj; Georgiou, Michalis; Mahroo, Omar A; Webster, Andrew R; Michaelides, Michel; Bakall, Benjamin; Iannaccone, Alessandro; Vincent, Ajoy; Parameswarappa, Deepika C; Heon, Elise; Scholl, Hendrik P N; Janeschitz-Kriegl, Lucas; Traboulsi, Elias I; Zein, Wadih; Brooks, Brian P; Cukras, Catherine; Hufnagel, Robert; Aleman, Tomas S; Sylla, Mohamed M; Tsang, Stephen H; Alabek, Michelle; Sahel, Jose; Gorin, Michael B; van Genderen, Maria M; Stingl, Katarina; Reith, Milda; Kohl, Susanne; Amaral, Rebeca Azevedo Souza; Sallum, Juliana Maria Ferraz; Vincent, Andrea L; Hull, Sarah; Duncan, Jacque L; Hanson, James V M; Tedeus, Matthias; Maggi, Jordi; Graf, Urs; Koller, Samuel; Berger, Wolfgang; Gerth-Kahlert, Christina; Marra, Molly; Everett, Lesley A; Yang, Paul; Pennesi, Mark E

Classification and Growth Rate of Chorioretinal Atrophy after Voretigene Neparvovec-Rzyl for RPE65-Mediated Retinal Degeneration

Voretigene Neparvovec-Rzyl治疗RPE65介导的视网膜变性后脉络膜视网膜萎缩的分类和生长速率

Bommakanti, Nikhil; Young, Benjamin K; Sisk, Robert A; Berrocal, Audina M; Duncan, Jacque L; Bakall, Benjamin; Mathias, Marc T; Ahmed, Ishrat; Chorfi, Sarah; Comander, Jason; Nagiel, Aaron; Besirli, Cagri G

Emerging Disease of the Desert: Rise of West Nile Virus Chorioretinitis in Arizona

沙漠新发疾病:亚利桑那州西尼罗河病毒性脉络膜视网膜炎病例激增

Odden, Jamie L; Patel, Dillan; Bryan, J Shepard; Bakall, Benjamin; Patel, Rima; De Souza, Stephen

Fully implanted battery-free high power platform for chronic spinal and muscular functional electrical stimulation.

用于慢性脊髓和肌肉功能性电刺激的完全植入式无电池高功率平台

Burton Alex, Wang Zhong, Song Dan, Tran Sam, Hanna Jessica, Ahmad Dhrubo, Bakall Jakob, Clausen David, Anderson Jerry, Peralta Roberto, Sandepudi Kirtana, Benedetto Alex, Yang Ethan, Basrai Diya, Miller Lee E, Tresch Matthew C, Gutruf Philipp

Wireless, fully implantable cardiac stimulation and recording with on-device computation for closed-loop pacing and defibrillation

无线、完全植入式心脏刺激和记录装置,具有用于闭环起搏和除颤的设备内计算功能

Ausra, Jokubas; Madrid, Micah; Yin, Rose T; Hanna, Jessica; Arnott, Suzanne; Brennan, Jaclyn A; Peralta, Roberto; Clausen, David; Bakall, Jakob A; Efimov, Igor R; Gutruf, Philipp

A Phase I, Single Ascending Dose Study of GEM103 (Recombinant Human Complement Factor H) in Patients with Geographic Atrophy

一项针对地图状萎缩患者的 GEM103(重组人补体因子 H)的 I 期单次递增剂量研究

Khanani, Arshad M; Maturi, Raj K; Bagheri, Nika; Bakall, Benjamin; Boyer, David S; Couvillion, Stephen S; Dhoot, Dilsher S; Holekamp, Nancy M; Jamal, Karim N; Marcus, Dennis M; Pieramici, Dante; Aziz, Aamir A; Patki, Kiran C; Bridges, William Z Jr; Barone, Samuel B

Characterization of the Spectrum of Ophthalmic Changes in Patients With Alagille Syndrome

阿拉吉尔综合征患者眼部改变谱的特征描述

da Palma, Mariana Matioli; Igelman, Austin D; Ku, Cristy; Burr, Amanda; You, Jia Yue; Place, Emily M; Wang, Nan-Kai; Oh, Jin Kyun; Branham, Kari E; Zhang, Xinxin; Ahn, Jeeyun; Gorin, Michael B; Lam, Byron L; Ronquillo, Cecinio C; Bernstein, Paul S; Nagiel, Aaron; Huckfeldt, Rachel; Cabrera, Michelle T; Kelly, John P; Bakall, Benjamin; Iannaccone, Alessandro; Hufnagel, Robert B; Zein, Wadih M; Koenekoop, Robert K; Birch, David G; Yang, Paul; Fahim, Abigail T; Pennesi, Mark E

Reduced penetrance in a large Caucasian pedigree with Stickler syndrome

在一个患有斯蒂克勒综合征的大型白种人家系中,外显率降低

Tompson, Stuart W; Johnson, Charles; Abbott, Diana; Bakall, Benjamin; Soler, Vincent; Yanovitch, Tammy L; Whisenhunt, Kristina N; Klemm, Thomas; Rozen, Steve; Stone, Edwin M; Johnson, Max; Young, Terri L

Human genome meeting 2016 : Houston, TX, USA. 28 February - 2 March 2016

2016年人类基因组会议:美国德克萨斯州休斯顿,2016年2月28日至3月2日

Srivastava, A K; Wang, Y; Huang, R; Skinner, C; Thompson, T; Pollard, L; Wood, T; Luo, F; Stevenson, R; Polimanti, R; Gelernter, J; Lin, X; Lim, I Y; Wu, Y; Teh, A L; Chen, L; Aris, I M; Soh, S E; Tint, M T; MacIsaac, J L; Yap, F; Kwek, K; Saw, S M; Kobor, M S; Meaney, M J; Godfrey, K M; Chong, Y S; Holbrook, J D; Lee, Y S; Gluckman, P D; Karnani, N; Kapoor, A; Lee, D; Chakravarti, A; Maercker, C; Graf, F; Boutros, M; Stamoulis, G; Santoni, F; Makrythanasis, P; Letourneau, A; Guipponi, M; Panousis, N; Garieri, M; Ribaux, P; Falconnet, E; Borel, C; Antonarakis, S E; Kumar, S; Curran, J; Blangero, J; Chatterjee, S; Kapoor, A; Akiyama, J; Auer, D; Berrios, C; Pennacchio, L; Chakravarti, A; Donti, T R; Cappuccio, G; Miller, M; Atwal, P; Kennedy, A; Cardon, A; Bacino, C; Emrick, L; Hertecant, J; Baumer, F; Porter, B; Bainbridge, M; Bonnen, P; Graham, B; Sutton, R; Sun, Q; Elsea, S; Hu, Z; Wang, P; Zhu, Y; Zhao, J; Xiong, M; Bennett, David A; Hidalgo-Miranda, A; Romero-Cordoba, S; Rodriguez-Cuevas, S; Rebollar-Vega, R; Tagliabue, E; Iorio, M; D’Ippolito, E; Baroni, S; Kaczkowski, B; Tanaka, Y; Kawaji, H; Sandelin, A; Andersson, R; Itoh, M; Lassmann, T; Hayashizaki, Y; Carninci, P; Forrest, A R R; Semple, C A; Rosenthal, E A; Shirts, B; Amendola, L; Gallego, C; Horike-Pyne, M; Burt, A; Robertson, P; Beyers, P; Nefcy, C; Veenstra, D; Hisama, F; Bennett, R; Dorschner, M; Nickerson, D; Smith, J; Patterson, K; Crosslin, D; Nassir, R; Zubair, N; Harrison, T; Peters, U; Jarvik, G; Menghi, F; Inaki, K; Woo, X; Kumar, P; Grzeda, K; Malhotra, A; Kim, H; Ucar, D; Shreckengast, P; Karuturi, K; Keck, J; Chuang, J; Liu, E T; Ji, B; Tyler, A; Ananda, G; Carter, G; Nikbakht, H; Montagne, M; Zeinieh, M; Harutyunyan, A; Mcconechy, M; Jabado, N; Lavigne, P; Majewski, J; Goldstein, J B; Overman, M; Varadhachary, G; Shroff, R; Wolff, R; Javle, M; Futreal, A; Fogelman, D; Bravo, L; Fajardo, W; Gomez, H; Castaneda, C; Rolfo, C; Pinto, J A; Akdemir, K C; Chin, L; Futreal, A; Patterson, S; Statz, C; Mockus, S; Nikolaev, S N; Bonilla, X I; Parmentier, L; King, B; Bezrukov, F; Kaya, G; Zoete, V; Seplyarskiy, V; Sharpe, H; McKee, T; Letourneau, A; Ribaux, P; Popadin, K; Basset-Seguin, N; Chaabene, R Ben; Santoni, F; Andrianova, M; Guipponi, M; Garieri, M; Verdan, C; Grosdemange, K; Sumara, O; Eilers, M; Aifantis, I; Michielin, O; de Sauvage, F; Antonarakis, S; Likhitrattanapisal, S; Lincoln, S; Kurian, A; Desmond, A; Yang, S; Kobayashi, Y; Ford, J; Ellisen, L; Peters, T L; Alvarez, K R; Hollingsworth, E F; Lopez-Terrada, D H; Hastie, A; Dzakula, Z; Pang, A W; Lam, E T; Anantharaman, T; Saghbini, M; Cao, H; Gonzaga-Jauregui, C; Ma, L; King, A; Rosenzweig, E Berman; Krishnan, U; Reid, J G; Overton, J D; Dewey, F; Chung, W K; Small, K; DeLuca, A; Cremers, F; Lewis, R A; Puech, V; Bakall, B; Silva-Garcia, R; Rohrschneider, K; Leys, M; Shaya, F S; Stone, E; Sobreira, N L; Schiettecatte, F; Ling, H; Pugh, E; Witmer, D; Hetrick, K; Zhang, P; Doheny, K; Valle, D; Hamosh, A; Jhangiani, S N; Akdemir, Z Coban; Bainbridge, M N; Charng, W; Wiszniewski, W; Gambin, T; Karaca, E; Bayram, Y; Eldomery, M K; Posey, J; Doddapaneni, H; Hu, J; Sutton, V R; Muzny, D M; Boerwinkle, E A; Valle, D; Lupski, J R; Gibbs, R A; Shekar, S; Salerno, W; English, A; Mangubat, A; Bruestle, J; Thorogood, A; Knoppers, B M; Takahashi, H; Nitta, K R; Kozhuharova, A; Suzuki, A M; Sharma, H; Cotella, D; Santoro, C; Zucchelli, S; Gustincich, S; Carninci, P; Mulvihill, J J; Baynam, G; Gahl, W; Groft, S C; Kosaki, K; Lasko, P; Melegh, B; Taruscio, D; Ghosh, R; Plon, S; Scherer, S; Qin, X; Sanghvi, R; Walker, K; Chiang, T; Muzny, D; Wang, L; Black, J; Boerwinkle, E; Weinshilboum, R; Gibbs, R; Karpinets, T; Calderone, T; Wani, K; Yu, X; Creasy, C; Haymaker, C; Forget, M; Nanda, V; Roszik, J; Wargo, J; Haydu, L; Song, X; Lazar, A; Gershenwald, J; Davies, M; Bernatchez, C; Zhang, J; Futreal, A; Woodman, S; Chesler, E J; Reynolds, T; Bubier, J A; Phillips, C; Langston, M A; Baker, E J; Xiong, M; Ma, L; Lin, N; Amos, C; Lin, N; Wang, P; Zhu, Y; Zhao, J; Calhoun, V; Xiong, M; Dobretsberger, O; Egger, M; Leimgruber, F; Sadedin, S; Oshlack, A; Antonio, V A A; Ono, N; Ahmed, Z; Bolisetty, M; Zeeshan, S; Anguiano, E; Ucar, D; Sarkar, A; Nandineni, M R; Zeng, C; Shao, J; Cao, H; Hastie, A; Pang, A W; Lam, E T; Liang, T; Pham, K; Saghbini, M; Dzakula, Z; Chee-Wei, Y; Dongsheng, L; Lai-Ping, W; Lian, D; Hee, R O Twee; Yunus, Y; Aghakhanian, F; Mokhtar, S S; Lok-Yung, C V; Bhak, J; Phipps, M; Shuhua, X; Yik-Ying, T; Kumar, V; Boon-Peng, H; Campbell, I; Young, M -A; James, P; Rain, M; Mohammad, G; Kukreti, R; Pasha, Q; Akilzhanova, A R; Guelly, C; Abilova, Z; Rakhimova, S; Akhmetova, A; Kairov, U; Trajanoski, S; Zhumadilov, Z; Bekbossynova, M; Schumacher, C; Sandhu, S; Harkins, T; Makarov, V; Doddapaneni, H; Glenn, R; Momin, Z; Dilrukshi, B; Chao, H; Meng, Q; Gudenkauf, B; Kshitij, R; Jayaseelan, J; Nessner, C; Lee, S; Blankenberg, K; Lewis, L; Hu, J; Han, Y; Dinh, H; Jireh, S; Walker, K; Boerwinkle, E; Muzny, D; Gibbs, R; Hu, J; Walker, K; Buhay, C; Liu, X; Wang, Q; Sanghvi, R; Doddapaneni, H; Ding, Y; Veeraraghavan, N; Yang, Y; Boerwinkle, E; Beaudet, A L; Eng, C M; Muzny, D M; Gibbs, R A; Worley, K C C; Liu, Y; Hughes, D S T; Murali, S C; Harris, R A; English, A C; Qin, X; Hampton, O A; Larsen, P; Beck, C; Han, Y; Wang, M; Doddapaneni, H; Kovar, C L; Salerno, W J; Yoder, A; Richards, S; Rogers, J; Lupski, J R; Muzny, D M; Gibbs, R A; Meng, Q; Bainbridge, M; Wang, M; Doddapaneni, H; Han, Y; Muzny, D; Gibbs, R; Harris, R A; Raveenedran, M; Xue, C; Dahdouli, M; Cox, L; Fan, G; Ferguson, B; Hovarth, J; Johnson, Z; Kanthaswamy, S; Kubisch, M; Platt, M; Smith, D; Vallender, E; Wiseman, R; Liu, X; Below, J; Muzny, D; Gibbs, R; Yu, F; Rogers, J; Lin, J; Zhang, Y; Ouyang, Z; Moore, A; Wang, Z; Hofmann, J; Purdue, M; Stolzenberg-Solomon, R; Weinstein, S; Albanes, D; Liu, C S; Cheng, W L; Lin, T T; Lan, Q; Rothman, N; Berndt, S; Chen, E S; Bahrami, H; Khoshzaban, A; Keshal, S Heidari; Bahrami, H; Khoshzaban, A; Keshal, S Heidari; Alharbi, K K R; Zhalbinova, M; Akilzhanova, A; Rakhimova, S; Bekbosynova, M; Myrzakhmetova, S; Matar, M; Mili, N; Molinari, R; Ma, Y; Guerrier, S; Elhawary, N; Tayeb, M; Bogari, N; Qotb, N; McClymont, S A; Hook, P W; Goff, L A; McCallion, A; Kong, Y; Charette, J R; Hicks, W L; Naggert, J K; Zhao, L; Nishina, P M; Edrees, B M; Athar, M; Al-Allaf, F A; Taher, M M; Khan, W; Bouazzaoui, A; Harbi, N A; Safar, R; Al-Edressi, H; Anazi, A; Altayeb, N; Ahmed, M A; Alansary, K; Abduljaleel, Z; Kratz, A; Beguin, P; Poulain, S; Kaneko, M; Takahiko, C; Matsunaga, A; Kato, S; Suzuki, A M; Bertin, N; Lassmann, T; Vigot, R; Carninci, P; Plessy, C; Launey, T; Graur, D; Lee, D; Kapoor, A; Chakravarti, A; Friis-Nielsen, J; Izarzugaza, J M; Brunak, S; Chakraborty, A; Basak, J; Mukhopadhyay, A; Soibam, B S; Das, D; Biswas, N; Das, S; Sarkar, S; Maitra, A; Panda, C; Majumder, P; Morsy, H; Gaballah, A; Samir, M; Shamseya, M; Mahrous, H; Ghazal, A; Arafat, W; Hashish, M; Gruber, J J; Jaeger, N; Snyder, M; Patel, K; Bowman, S; Davis, T; Kraushaar, D; Emerman, A; Russello, S; Henig, N; Hendrickson, C; Zhang, K; Rodriguez-Dorantes, M; Cruz-Hernandez, C D; Garcia-Tobilla, C D P; Solorzano-Rosales, S; Jäger, N; Chen, J; Haile, R; Hitchins, M; Brooks, J D; Snyder, M; Jiménez-Morales, S; Ramírez, M; Nuñez, J; Bekker, V; Leal, Y; Jiménez, E; Medina, A; Hidalgo, A; Mejía, J; Halytskiy, V; Naggert, J; Collin, G B; DeMauro, K; Hanusek, R; Nishina, P M; Belhassa, K; Belhassan, K; Bouguenouch, L; Samri, I; Sayel, H; moufid, FZ; El Bouchikhi, I; Trhanint, S; Hamdaoui, H; Elotmani, I; Khtiri, I; Kettani, O; Quibibo, L; Ahagoud, M; Abbassi, M; Ouldim, K; Marusin, A V; Kornetov, A N; Swarovskaya, M; Vagaiceva, K; Stepanov, V; De La Paz, E M Cutiongco; Sy, R; Nevado, J; Reganit, P; Santos, L; Magno, J D; Punzalan, F E; Ona, D; Llanes, E; Santos-Cortes, R L; Tiongco, R; Aherrera, J; Abrahan, L; Pagauitan-Alan, P; Morelli, K H; Domire, J S; Pyne, N; Harper, S; Burgess, R; Zhalbinova, M; Akilzhanova, A; Rakhimova, S; Bekbosynova, M; Myrzakhmetova, S; Gari, M A; Dallol, A; Alsehli, H; Gari, A; Gari, M; Abuzenadah, A; Thomas, M; Sukhai, M; Garg, S; Misyura, M; Zhang, T; Schuh, A; Stockley, T; Kamel-Reid, S; Sherry, S; Xiao, C; Slotta, D; Rodarmer, K; Feolo, M; Kimelman, M; Godynskiy, G; O’Sullivan, C; Yaschenko, E; Xiao, C; Yaschenko, E; Sherry, S; Rangel-Escareño, C; Rueda-Zarate, H; Tayubi, I A; Mohammed, R; Ahmed, I; Ahmed, T; Seth, S; Amin, S; Song, X; Mao, X; Sun, H; Verhaak, R G; Futreal, A; Zhang, J; Whiite, S J; Chiang, T; English, A; Farek, J; Kahn, Z; Salerno, W; Veeraraghavan, N; Boerwinkle, E; Gibbs, R; Kasukawa, T; Lizio, M; Harshbarger, J; Hisashi, S; Severin, J; Imad, A; Sahin, S; Freeman, T C; Baillie, K; Sandelin, A; Carninci, P; Forrest, A R R; Kawaji, H; Salerno, W; English, A; Shekar, S N; Mangubat, A; Bruestle, J; Boerwinkle, E; Gibbs, R A; Salem, A H; Ali, M; Ibrahim, A; Ibrahim, M; Barrera, H A; Garza, L; Torres, J A; Barajas, V; Ulloa-Aguirre, A; Kershenobich, D; Mortaji, Shahroj; Guizar, Pedro; Loera, Eliezer; Moreno, Karen; De León, Adriana; Monsiváis, Daniela; Gómez, Jackeline; Cardiel, Raquel; Fernandez-Lopez, J C; Bonifaz-Peña, V; Rangel-Escareño, C; Hidalgo-Miranda, A; Contreras, A V; Polfus, L; Wang, X; Philip, V; Carter, G; Abuzenadah, A A; Gari, M; Turki, R; Dallol, A; Uyar, A; Kaygun, A; Zaman, S; Marquez, E; George, J; Ucar, D; Hendrickson, C L; Emerman, A; Kraushaar, D; Bowman, S; Henig, N; Davis, T; Russello, S; Patel, K; Starr, D B; Baird, M; Kirkpatrick, B; Sheets, K; Nitsche, R; Prieto-Lafuente, L; Landrum, M; Lee, J; Rubinstein, W; Maglott, D; Thavanati, P K R; de Dios, A Escoto; Hernandez, R E Navarro; Aldrate, M E Aguilar; Mejia, M R Ruiz; Kanala, K R R; Abduljaleel, Z; Khan, W; Al-Allaf, F A; Athar, M; Taher, M M; Shahzad, N; Bouazzaoui, A; Huber, E; Dan, A; Al-Allaf, F A; Herr, W; Sprotte, G; Köstler, J; Hiergeist, A; Gessner, A; Andreesen, R; Holler, E; Al-Allaf, F; Alashwal, A; Abduljaleel, Z; Taher, M; Bouazzaoui, A; Abalkhail, H; Al-Allaf, A; Bamardadh, R; Athar, M; Filiptsova, O; Kobets, M; Kobets, Y; Burlaka, I; Timoshyna, I; Filiptsova, O; Kobets, M N; Kobets, Y; Burlaka, I; Timoshyna, I; Filiptsova, O; Kobets, M N; Kobets, Y; Burlaka, I; Timoshyna, I; Al-allaf, F A; Mohiuddin, M T; Zainularifeen, A; Mohammed, A; Abalkhail, H; Owaidah, T; Bouazzaoui, A