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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials
Maguire, Albert M, Russell, Stephen, Wellman, Jennifer A, Chung, Daniel C, Yu, Zi-Fan, Tillman, Amy, Wittes, Janet, Pappas, Julie, Elci, Okan, Marshall, Kathleen A, McCague, Sarah, Reichert, Hannah, Davis, Maria, Simonelli, Francesca, Leroy, Bart P, Wright, J Fraser, High, Katherine A, Bennett, Jean
Published in Ophthalmology (Rochester, Minn.) (01.09.2019)
Published in Ophthalmology (Rochester, Minn.) (01.09.2019)
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Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
Fitzgerald, Kevin, Frank-Kamenetsky, Maria, Shulga-Morskaya, Svetlana, Liebow, Abigail, Bettencourt, Brian R, Sutherland, Jessica E, Hutabarat, Renta M, Clausen, Valerie A, Karsten, Verena, Cehelsky, Jeffrey, Nochur, Saraswathy V, Kotelianski, Victor, Horton, Jay, Mant, Timothy, Chiesa, Joseph, Ritter, James, Munisamy, Malathy, Vaishnaw, Akshay K, Gollob, Jared A, Simon, Amy
Published in The Lancet (British edition) (04.01.2014)
Published in The Lancet (British edition) (04.01.2014)
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Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial
Warren Olanow, C., Bartus, Raymond T., Baumann, Tiffany L., Factor, Stewart, Boulis, Nicholas, Stacy, Mark, Turner, Dennis A., Marks, William, Larson, Paul, Starr, Phillip A., Jankovic, Joseph, Simpson, Richard, Watts, Ray, Guthrie, Barton, Poston, Kathleen, Henderson, Jaimie M., Stern, Matthew, Baltuch, Gordon, Goetz, Christopher G., Herzog, Christopher, Kordower, Jeffrey H., Alterman, Ron, Lozano, Andres M., Lang, Anthony E.
Published in Annals of neurology (01.08.2015)
Published in Annals of neurology (01.08.2015)
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Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial
Mishra, Alaknanda, Vijayasarathy, Camasamudram, Cukras, Catherine A., Wiley, Henry E., Sen, H. Nida, Zeng, Yong, Wei, Lisa L., Sieving, Paul A.
Published in Molecular therapy (02.06.2021)
Published in Molecular therapy (02.06.2021)
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Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy
Weleber, Richard G., Pennesi, Mark E., Wilson, David J., Kaushal, Shalesh, Erker, Laura R., Jensen, Lauren, McBride, Maureen T., Flotte, Terence R., Humphries, Margaret, Calcedo, Roberto, Hauswirth, William W., Chulay, Jeffrey D., Stout, J. Timothy
Published in Ophthalmology (Rochester, Minn.) (01.07.2016)
Published in Ophthalmology (Rochester, Minn.) (01.07.2016)
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Phenotypic Characterization of Genetically Lowered Human Lipoprotein(a) Levels
Emdin, Connor A., Khera, Amit V., Natarajan, Pradeep, Klarin, Derek, Won, Hong-Hee, Peloso, Gina M., Stitziel, Nathan O., Nomura, Akihiro, Zekavat, Seyedeh M., Bick, Alexander G., Gupta, Namrata, Asselta, Rosanna, Duga, Stefano, Merlini, Piera Angelica, Correa, Adolfo, Kessler, Thorsten, Wilson, James G., Bown, Matthew J., Hall, Alistair S., Braund, Peter S., Samani, Nilesh J., Schunkert, Heribert, Marrugat, Jaume, Elosua, Roberto, McPherson, Ruth, Farrall, Martin, Watkins, Hugh, Willer, Cristen, Abecasis, Gonçalo R., Felix, Janine F., Vasan, Ramachandran S., Lander, Eric, Rader, Daniel J., Danesh, John, Ardissino, Diego, Gabriel, Stacey, Saleheen, Danish, Kathiresan, Sekar
Published in Journal of the American College of Cardiology (27.12.2016)
Published in Journal of the American College of Cardiology (27.12.2016)
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Magnetic resonance imaging–guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease
Christine, Chadwick W., Bankiewicz, Krystof S., Van Laar, Amber D., Richardson, R. Mark, Ravina, Bernard, Kells, Adrian P., Boot, Brendon, Martin, Alastair J., Nutt, John, Thompson, Marin E., Larson, Paul S.
Published in Annals of neurology (01.05.2019)
Published in Annals of neurology (01.05.2019)
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Therapeutic nucleic acids: current clinical status
Sridharan, Kannan, Gogtay, Nithya Jaideep
Published in British journal of clinical pharmacology (01.09.2016)
Published in British journal of clinical pharmacology (01.09.2016)
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Lymphoid Regeneration from Gene-Corrected SCID-X1 Subject-Derived iPSCs
Menon, Tushar, Firth, Amy L., Scripture-Adams, Deirdre D., Galic, Zoran, Qualls, Susan J., Gilmore, William B., Ke, Eugene, Singer, Oded, Anderson, Leif S., Bornzin, Alexander R., Alexander, Ian E., Zack, Jerome A., Verma, Inder M.
Published in Cell stem cell (02.04.2015)
Published in Cell stem cell (02.04.2015)
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Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans
Candotti, Fabio, Shaw, Kit L., Muul, Linda, Carbonaro, Denise, Sokolic, Robert, Choi, Christopher, Schurman, Shepherd H., Garabedian, Elizabeth, Kesserwan, Chimene, Jagadeesh, G. Jayashree, Fu, Pei-Yu, Gschweng, Eric, Cooper, Aaron, Tisdale, John F., Weinberg, Kenneth I., Crooks, Gay M., Kapoor, Neena, Shah, Ami, Abdel-Azim, Hisham, Yu, Xiao-Jin, Smogorzewska, Monika, Wayne, Alan S., Rosenblatt, Howard M., Davis, Carla M., Hanson, Celine, Rishi, Radha G., Wang, Xiaoyan, Gjertson, David, Yang, Otto O., Balamurugan, Arumugam, Bauer, Gerhard, Ireland, Joanna A., Engel, Barbara C., Podsakoff, Gregory M., Hershfield, Michael S., Blaese, R. Michael, Parkman, Robertson, Kohn, Donald B.
Published in Blood (01.11.2012)
Published in Blood (01.11.2012)
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Gene Therapy for Inherited Retinal Disease: Long-Term Durability of Effect
Leroy, Bart P., Fischer, M. Dominik, Flannery, John G., MacLaren, Robert E., Dalkara, Deniz, Scholl, Hendrik P.N., Chung, Daniel C., Spera, Claudio, Viriato, Daniel, Banhazi, Judit
Published in Ophthalmic research (01.01.2023)
Published in Ophthalmic research (01.01.2023)
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Phase I study of liver depot gene therapy in late-onset Pompe disease
Smith, Edward C., Hopkins, Sam, Case, Laura E., Xu, Ming, Walters, Crista, Dearmey, Stephanie, Han, Sang-oh, Spears, Tracy G., Chichester, Jessica A., Bossen, Edward H., Hornik, Christoph P., Cohen, Jennifer L., Bali, Deeksha, Kishnani, Priya S., Koeberl, Dwight D.
Published in Molecular therapy (05.07.2023)
Published in Molecular therapy (05.07.2023)
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Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial
Ghazi, Nicola G., Abboud, Emad B., Nowilaty, Sawsan R., Alkuraya, Hisham, Alhommadi, Abdulrahman, Cai, Huimin, Hou, Rui, Deng, Wen-Tao, Boye, Sanford L., Almaghamsi, Abdulrahman, Al Saikhan, Fahad, Al-Dhibi, Hassan, Birch, David, Chung, Christopher, Colak, Dilek, LaVail, Matthew M., Vollrath, Douglas, Erger, Kirsten, Wang, Wenqiu, Conlon, Thomas, Zhang, Kang, Hauswirth, William, Alkuraya, Fowzan S.
Published in Human genetics (01.03.2016)
Published in Human genetics (01.03.2016)
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Antisense Oligonucleotide Inhibition of Apolipoprotein C-III Reduces Plasma Triglycerides in Rodents, Nonhuman Primates, and Humans
Graham, Mark J., Lee, Richard G., Bell, Thomas A., Fu, Wuxia, Mullick, Adam E., Alexander, Veronica J., Singleton, Walter, Viney, Nick, Geary, Richard, Su, John, Baker, Brenda F., Burkey, Jennifer, Crooke, Stanley T., Crooke, Rosanne M.
Published in Circulation research (24.05.2013)
Published in Circulation research (24.05.2013)
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Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial
Somanathan, Suryanarayan, Calcedo, Roberto, Wilson, James M.
Published in Molecular therapy (04.03.2020)
Published in Molecular therapy (04.03.2020)
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Superior Retinal Gene Transfer and Biodistribution Profile of Subretinal Versus Intravitreal Delivery of AAV8 in Nonhuman Primates
Seitz, Immanuel P., Michalakis, Stylianos, Wilhelm, Barbara, Reichel, Felix F., Ochakovski, G. Alex, Zrenner, Eberhart, Ueffing, Marius, Biel, Martin, Wissinger, Bernd, Bartz-Schmidt, Karl U., Peters, Tobias, Fischer, M. Dominik
Published in Investigative ophthalmology & visual science (01.11.2017)
Published in Investigative ophthalmology & visual science (01.11.2017)
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Changes in ventricular remodelling and clinical status during the year following a single administration of stromal cell-derived factor-1 non-viral gene therapy in chronic ischaemic heart failure patients: the STOP-HF randomized Phase II trial
Chung, Eugene S., Miller, Leslie, Patel, Amit N., Anderson, Russell David, Mendelsohn, Farrell O., Traverse, Jay, Silver, Kevin H., Shin, Julia, Ewald, Gregory, Farr, Mary Jane, Anwaruddin, Saif, Plat, Francis, Fisher, Scott J., AuWerter, Alexander T., Pastore, Joseph M., Aras, Rahul, Penn, Marc S.
Published in European heart journal (01.09.2015)
Published in European heart journal (01.09.2015)
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Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells
De Ravin, Suk See, Brault, Julie, Meis, Ronald J., Liu, Siyuan, Li, Linhong, Pavel-Dinu, Mara, Lazzarotto, Cicera R., Liu, Taylor, Koontz, Sherry M., Choi, Uimook, Sweeney, Colin L., Theobald, Narda, Lee, GaHyun, Clark, Aaron B., Burkett, Sandra S., Kleinstiver, Benjamin P., Porteus, Matthew H., Tsai, Shengdar, Kuhns, Douglas B., Dahl, Gary A., Headey, Stephen, Wu, Xiaolin, Malech, Harry L.
Published in Blood (13.05.2021)
Published in Blood (13.05.2021)
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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
Mendell, Jerry R, Sahenk, Zarife, Malik, Vinod, Gomez, Ana M, Flanigan, Kevin M, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Meadows, Eric, Lewis, Sarah, Braun, Lyndsey, Shontz, Kim, Rouhana, Maria, Clark, Kelly Reed, Rosales, Xiomara Q, Al-Zaidy, Samiah, Govoni, Alessandra, Rodino-Klapac, Louise R, Hogan, Mark J, Kaspar, Brian K
Published in Molecular therapy (01.01.2015)
Published in Molecular therapy (01.01.2015)
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