Fragile X syndrome gene therapy could be safer with an FMRP “safety dial” that self-regulates expression using natural FMR1 control elements.
With $89K from FRAXA, Dr. David Bloom’s team advanced Fragile X gene therapy by transferring the fmr1 gene into mouse brains via viral vectors.
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FRAXA Funded Research
Current Research Grants (38)
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Select ResearcherAbolghasemi, Armita (1)Acharya, Anirudh (1)Albeck, David (1)Alger, Bradley (1)Allada, Ravi (1)Allen, Nicola (1)Annear, Dale (2)Auerbach Benjamin (1)Bacigalupo, Juan (1)Bagni, Claudia (3)Bailey, Don (2)Banerjee, Probal (1)Banke, Tue (2)Bardoni, Barbara (6)Barnes, Stephanie (3)Bassell, Gary (3)Bauchwitz, Robert (1)Bear, Mark (16)Beasley, Lindsay (1)Bergdolt, Lara (1)Berry-Kravis, Elizabeth (17)Bettio, Luis (1)Bhakar, Asha (1)Bhattacharya, Aditi (2)Bhattacharyya, Anita (5)Bilousova, Tina (1)Binder, Devin (4)Bloom, David (1)Borreca, Antonella (4)Bosch, Miquel (1)Boussadia, Zaira (2)Brager, Darrin (1)Braun, Katarina (1)Brenman, Jay (1)Brennan, Francis (1)Briault, Sylvain (3)Broadie, Kendal (1)Brown, W Ted (1)Budylin, Tatyana (1)Caku, Artuela (2)Canal, Clinton E. (4)Cancedda, Laura (1)Carthew, Richard (1)Carullo, Paulina (2)Castren, Maija (1)Cea-Del Rio, Christian (1)Ceman, Stephanie (1)Chater, Dounia (1)Chattarji, Sumantra (3)Chen, Chloe (2)Cheng, Ning (1)Choi, Catherine (3)Christie, Brian (2)Ciranna, Lucia (1)Cline, Holly (1)Cogram, Patricia (8)Contractor, Anis (2)Corbin, Francois (3)Corbin, Joshua (1)Coulson, Rochelle (2)Cowan, Christopher (1)Cowan, Justin (1)Cox, Charles (1)Crnic, Linda (1)Croom, Katilynne (1)D’Ercole, Martina (1)Darnell, Jennifer (1)Darnell, Robert (1)Davidovic, Laetitia (1)Davidson, Beverly (1)de la Torre, Rafael (2)De Stefano, Lisa (1)de Vrij, Femke (1)Deacon, Robert (2)Dempsey, Graham (1)Deng, James (1)Deng, Pan-Yue (1)Denman, Robert (2)Di Marino, Daniele (1)Dierssen, Mara (2)Dijck, Anke Van (2)Dionne, Olivier (1)Disney, Matthew (1)Dobkin, Carl (1)Dockendorff, Thomas (1)Doering, Laurie (1)Dumontier, Dimitri (1)Dunaevsky, Anna (1)Dykens, Elisabeth (1)El Idrissi, Abdeslem (1)El-Osta, Assam (1)Elvassore, Nicola (1)Erickson, Craig (14)Ethell, Iryna (6)Fallon, Justin (2)Feng, Jia (1)Feng, Yue (1)Fink, James (1)Finucane, Brenda (1)Francesconi, Anna (1)Frick, Andreas (2)Galera-López, Lorena (1)Gan, Wen-Biao (2)Gantois, Ilse (2)Gao, Fen-Biao (1)Gao, Pan (1)Garkun, Yury (1)Gatto, Cheryl (1)Gelfand, Vladimir (1)Geoffroy, Andrea (1)Germain, Noelle (1)Giangrande, Angela (1)Gibson, Jay (2)Goncalves, Tiago (1)Greenough, William (1)Grokoski, Kamila Castro (1)Gross, Christina (2)Guiotto, Annamaria (1)Guo, Ziyuan (1)Gurney, Mark (2)Gutierrez, Alejandra (1)Ha, Seong Eon (1)Haenfler, Jill (1)Hagerman, Paul (1)Hagerman, Randi (2)Haggarty, Stephen (1)Hassan, Bassem (1)He, Qionger (2)Heaney, Chelcie (1)Hessl, David (1)Higashimori, Haruki (1)Hogan, Carolyn Ann (1)Holley, Andrew (1)Hoogeveen, Andre (1)Huber, Kimberly (8)Huganier, Richard (1)Huntsman, Molly (2)Inamdar, Manasi (1)Ingolia, Nicholas (1)Jaffrey, Samie (3)Jiang, Yong-Hui (1)Jin, Peng (5)Johnson-Glenberg, Mina (1)Johnston, Daniel (2)Jonak, Carrie (1)Jonas, Elizabeth (3)Jongens, Tom (4)Jope, Richard (1)Joyce, Karen (1)Kaczmarek, Leonard (7)Kandel, Eric (1)Kanellpoulos, Alexandros (1)Kang, Seo-Jun (1)Kang, Yunhee (2)Kaufmann, Walter (1)Khandjian, Edouard (1)Kim, Hwan (1)Kim, J. Wren (1)Kind, Peter (2)Kindler, Stephan (1)King, Margaret (2)Klann, Eric (2)Klug, Achim (1)Klyachko, Vitaly (3)Koeglsperger, Thomas (1)Kooy, Frank (8)Kornfeld-Sylla, Sara (1)Kosti, Adam (1)Kourdougli Nazim (1)Kreienkamp, Hans-Jurgen (1)Krzisch Marine Anais (1)Kumar, Vikas (1)Kumar, Vipendra (1)Kumari, Daman (1)Kurosaki, Tatsuaki (3)Larson, John (1)Lauder, Jean (1)Lauterborn, Julie (1)LeDoux, Joseph (1)Lee, Hungoo (2)Lee, Hye Young (1)Lee, Jeannie (10)Lepage, Jean-Francois (2)Levy, Richard (1)Li, Wenqi (1)Li, Xuekun (1)Licznerski, Pawel (1)Lieberman, David (1)Lombroso, Paul (1)Lovelace, Jonny (2)Lu, Lu (1)Mack, Kenneth (1)Malter, James (1)Mandel, Jean-Louis (1)Mansvelder, Huibert (1)Manzoni, Olivier (1)Maquat, Lynne (4)Marro, Samuele (1)Marsillo, Alexandra (1)Martin, Stéphane (1)Martire, Alberto (4)Mato Santos, Susana (1)Maurin, Thomas (3)McBride, Sean (4)McCamphill, Patrick (3)McCullagh, Elizabeth (1)McDougle, Christopher (1)McKenna, Mary (1)Meffert, Mollie (1)Men, Yuqin (1)Mihailescu, Mihaela (1)Moine, Herve (3)Morishita, Hirofumi (1)Morris, David (1)Moses, Kevin (1)Mourrain, Philippe Jacques (2)Moy, Sheryl (1)Muddashetty, Ravi (1)Murgia, Alessandra (1)Nelson, Charles A. (3)Nelson, David (3)Neri, Giovanni (3)Njem, Nadine (1)O'Malley, Karen (1)O’Brien, Alana (1)O’Donnell, Cian (1)Oh, Ji-eun (2)Oliviera Laura (1)Oostra, Ben (2)Osterweil, Emily (6)Ozaita, Andres (2)Paribello, Carlo (1)Paylor, Richard (1)Pedapati, Ernest (2)Pepper, Anita (1)Pichon, Jacques (2)Pirbhoy, Patricia (4)Portera-Cailliau, Carlos (2)Pouchelon, Gabrielle (1)Pouladi, Mahmoud (2)Pronot, Marie (1)Protic, Dragana (1)Raab-Graham, Kimberly (1)Rattazzi, Mario (1)Razak, Khaleel (7)Regan, Lynne (1)Reis, Surya (1)Renwick, Neil (1)Reynolds, Kathryn (1)Ribecco, Carmela (1)Richards, Robert (1)Richter, Joel (10)Romagnoli, Alice (1)Rotman, Ziv (1)Ruppert, Kathryn (1)Saraf, Tanishka (1)Sawacha, Zimi (1)Schaefer, Tori (1)Schmitt, Lauren (1)Scott, Angela (1)Segal, Menahem (1)Sejnowski, Terrence (1)Senter, Rebecca (2)Shah, Sneha (8)Shen, Minjie (1)Siegel, Jennifer (1)Silva, Alcino (1)Singh, Aditi (1)Siomi, Haruhiko (1)Siomi, Mikko (1)Sirois, Carissa (1)Skoulakis, Efthimios (1)Smith, Carolyn B. (4)Smith, Elizabeth (1)Smith, Laura N. (1)Smith, Stephen E.P. (1)Soderling, Scott (1)Sonenberg, Nahum (6)Spolaor, Fabiola (1)Stackhouse, Tracy (1)Stamenkovic, Vera (1)Stanfield, Andrew (1)Steward, Oswald (3)Svendsen, Clive (1)Svoboda, Karel (2)Tang, Weiping (1)Tartaglia, Nicole (1)Tartakoff, Alan (1)Tejada-Simons, MariVi (1)Tempio, Alessandra (1)Thacker, Jonathan (1)Tiedge, Henri (1)Todd, Peter (9)Tonegawa, Susumu (1)Toth, Miklos (2)Tran, Tien Phuoc (1)Tremblay, Marie-Eve (1)Tsai, Nien-Pei (2)Tsai, Yuan-Chen (1)Turner, Raymond (3)Tuschl, Thomas (1)Usdin, Karen (1)Utami, Kagistia (1)van der Lei, Mathijs (3)Vanderklish, Peter (7)Wang, Eric (1)Wang, Gordon (2)Wang, Hongbing (1)Warren, Stephen (2)Watanabe, Momoko (1)Weiler, Ivan Jeanne (1)Weisz, Eliana D. (1)Wen, Zhexing (4)Wernig, Marius (1)Westmark, Cara (5)Whitehead, Kathryn (1)Wilhelm, Michael (1)Wilkinson, Carol (4)Willemsen, Rob (1)Williams, Leena E. (1)Winograd, Claudia (1)Wong, Robert (1)Worley, Paul (1)Wright, Damien (1)Xu, Jie (1)Xu, Zhiyan (1)Yalan, Zhang (1)Yang, Wang-Yong (1)Yang, Yongjie (2)Yerneni, Saigopalakrishna (1)Yin, Jerry (1)Yrigollen, Carolyn (1)Yuskaitis, Christopher (1)Zang, Tong (1)Zhan, Xiaoqin (3)Zhao, Xinyu (7)Zhong, Jun (1)Zhu, Julius (2)Zoupi, Lida (1)Zukin, Suzanne (2)
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