Search Results - "Braun, Maurine"

Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models by Lee, Richard G., Mazzola, Anne Marie, Braun, Maurine C., Platt, Colin, Vafai, Scott B., Kathiresan, Sekar, Rohde, Ellen, Bellinger, Andrew M., Khera, Amit V.

“…VERVE-101 is an investigational in vivo CRISPR base-editing medicine designed to alter a single DNA base in the gene, permanently turn off hepatic protein…”
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In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates by Musunuru, Kiran, Chadwick, Alexandra C., Mizoguchi, Taiji, Garcia, Sara P., DeNizio, Jamie E., Reiss, Caroline W., Wang, Kui, Iyer, Sowmya, Dutta, Chaitali, Clendaniel, Victoria, Amaonye, Michael, Beach, Aaron, Berth, Kathleen, Biswas, Souvik, Braun, Maurine C., Chen, Huei-Mei, Colace, Thomas V., Ganey, John D., Gangopadhyay, Soumyashree A., Garrity, Ryan, Kasiewicz, Lisa N., Lavoie, Jennifer, Madsen, James A., Matsumoto, Yuri, Mazzola, Anne Marie, Nasrullah, Yusuf S., Nneji, Joseph, Ren, Huilan, Sanjeev, Athul, Shay, Madeleine, Stahley, Mary R., Fan, Steven H. Y., Tam, Ying K., Gaudelli, Nicole M., Ciaramella, Giuseppe, Stolz, Leslie E., Malyala, Padma, Cheng, Christopher J., Rajeev, Kallanthottathil G., Rohde, Ellen, Bellinger, Andrew M., Kathiresan, Sekar

“…Gene-editing technologies, which include the CRISPR–Cas nucleases 1 – 3 and CRISPR base editors 4 , 5 , have the potential to permanently modify…”
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Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models by Liu, Yuanjing, Dodart, Jean-Cosme, Tran, Helene, Berkovitch, Shaunna, Braun, Maurine, Byrne, Michael, Durbin, Ann F., Hu, Xiao Shelley, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Longo, Kenneth, Ruschel, Jörg, Shelke, Juili, Yang, Hailin, Yin, Yuan, Donner, Amy, Zhong, Zhong, Vargeese, Chandra, Brown, Robert H.

“…A large G 4 C 2 -repeat expansion in C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)…”
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Defective daily temperature regulation in a mouse model of amyotrophic lateral sclerosis by Braun, Maurine C., Castillo-Ruiz, Alexandra, Indic, Premananda, Jung, Dae Young, Kim, Jason K., Brown, Robert H., Swoap, Steven J., Schwartz, William J.

“…Current understanding of the pathogenesis of the familial form of amyotrophic lateral sclerosis has been aided by the study of transgenic mice that…”
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Kinematics of reaching and implications for handedness in rhesus monkey infants by Nelson, Eliza L., Konidaris, George D., Berthier, Neil E., Braun, Maurine C., Novak, Matthew F.S.X., Suomi, Stephen J., Novak, Melinda A.

“…Kinematic studies of reaching in human infants using two‐dimensional (2‐D) and three‐dimensional (3‐D) recordings have complemented behavioral studies of…”
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Abstract 11274: VERVE-101—An Investigational Single-Course Gene Editing Medicine Targeting PCSK9—Durably and Potently Lowers PCSK9 and LDL-C Concentrations in Non-Human Primates by Lee, Richard, Mazzola, Anne M, Braun, Maurine, Platt, Colin, Vafai, Scott, Kathiresan, Sekar, rohde, ellen, Bellinger, Andrew M, Khera, Amit V

“…IntroductionVERVE-101 is an in vivo CRISPR base editing therapy designed to alter a single DNA base in the PCSK9 gene, permanently inactivate hepatic…”
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An investigational in vivo base editing medicine targeting ANGPTL3, VERVE-201, achieves precise and durable liver editing in nonclinical studies by Lee, Richard, Mazzola, Anne, Denizio, Jamie, Mizoguchi, Taiji, Clendaniel, Victoria, Garrity, Ryan, Cox, Nicholas, Glass, Zachary, Chamarthi, Hareesh, Flannigan, Sydney, Matsumoto, Yuri, Braun, Maurine, Kathiresan, Sekar, Biedenkapp, Joseph, Rohde, Ellen, Bellinger, Andrew, Khera, Amit

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Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice by van Til, Niek P., Dogan, Yildirim, Barese, Cecilia, Unnisa, Zeenath, Guda, Swaroopa, Schindler, Rena, Yoon, John, Jacobs, Mary, Chiyyeadu, Abhishek, Pizzurro, Daniella, Liu, Bianling, Braun, Maurine, Oborski, Christine, Schambach, Axel, Harper, Claudia, Pfeifer, Richard, Mason, Chris

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Sa1465 - The Pharmacological Properties of APOC3 Antisense Oligonucleotides are Enhanced by Control of Stereochemistry by Apponi, Luciano, Iwamoto, Naoki, Frank-Kamenetsky, Maria, Yang, Hailin, Braun, Maurine, Shah, Anee, Yin, Yuan, Butler, David, Lu, Genliang, Menon, Sethu, Standley, Stephany, Aduda, Vincent, Bowman, Keith, Longo, Kenneth, Pittet, Lynnelle, Zhang, Jason, Zhong, Zhong, Vargeese, Chandra

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