Search Results - "Rohde, Ellen"

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing by Finn, Jonathan D., Smith, Amy Rhoden, Patel, Mihir C., Shaw, Lucinda, Youniss, Madeleine R., van Heteren, Jane, Dirstine, Tanner, Ciullo, Corey, Lescarbeau, Reynald, Seitzer, Jessica, Shah, Ruchi R., Shah, Aalok, Ling, Dandan, Growe, Jacqueline, Pink, Melissa, Rohde, Ellen, Wood, Kristy M., Salomon, William E., Harrington, William F., Dombrowski, Christian, Strapps, Walter R., Chang, Yong, Morrissey, David V.

“…The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome…”
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GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy by Kasiewicz, Lisa N., Biswas, Souvik, Beach, Aaron, Ren, Huilan, Dutta, Chaitali, Mazzola, Anne Marie, Rohde, Ellen, Chadwick, Alexandra, Cheng, Christopher, Garcia, Sara P., Iyer, Sowmya, Matsumoto, Yuri, Khera, Amit V., Musunuru, Kiran, Kathiresan, Sekar, Malyala, Padma, Rajeev, Kallanthottathil G., Bellinger, Andrew M.

“…Lipid nanoparticles have demonstrated utility in hepatic delivery of a range of therapeutic modalities and typically deliver their cargo via low-density…”
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Monoclonal antibody exposure in rat and cynomolgus monkey cerebrospinal fluid following systemic administration by Wang, Qin, Delva, Luisette, Weinreb, Paul H, Pepinsky, Robert B, Graham, Danielle, Veizaj, Elvana, Cheung, Anne E, Chen, Weiping, Nestorov, Ivan, Rohde, Ellen, Caputo, Robin, Kuesters, Geoffrey M, Bohnert, Tonika, Gan, Liang-Shang

“…Many studies have focused on the challenges of small molecule uptake across the blood-brain barrier, whereas few in-depth studies have assessed the challenges…”
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A stable IgG-like bispecific antibody targeting the epidermal growth factor receptor and the type I insulin-like growth factor receptor demonstrates superior anti-tumor activity by Dong, Jianying, Sereno, Arlene, Aivazian, Dikran, Langley, Emma, Miller, Brian R., Snyder, William B, Chan, Eric, Cantele, Matt, Morena, Ronald, Joseph, Ingrid B.J.K., Boccia, Antonio, Virata, Cyrus, Gamez, James, Yco, Grace, Favis, Michael, Wu, Xiufeng, Graff, Christilyn P., Wang, Qin, Rohde, Ellen, Rennard, Rachel, Berquist, Lisa, Huang, Flora, Zhang, Ying, Gao, Sharon X., Ho, Steffan N., Demarest, Stephen J., Reff, Mitchell E., Hariharan, Kandasamy, Glaser, Scott M.

“…The epidermal growth factor receptor (EGFR) and the type I insulin-like growth factor receptor (IGF-1R) are two cell surface receptor tyrosine kinases known to…”
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Stereochemistry–activity relationship of orally active tetralin S1P agonist prodrugs by Ma, Bin, Guckian, Kevin M., Lin, Edward Yin-Shiang, Lee, Wen-Cherng, Scott, Daniel, Kumaravel, Gnanasambandam, Macdonald, Timothy L., Lynch, Kevin R., Black, Cheryl, Chollate, Sowmya, Hahm, Kyungmin, Hetu, Gregg, Jin, Ping, Luo, Yi, Rohde, Ellen, Rossomando, Anthony, Scannevin, Robert, Wang, Joy, Yang, Chunhua

“…The synthesis, characterization and stereochemistry activity relationship of tetralin S1P agonist prodrugs is reported. (2 R,2′ S)- 5 (ED 50 = 0.1 mg/kg) was…”
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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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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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Protein analysis by membrane preconcentration–capillary electrophoresis: systematic evaluation of parameters affecting preconcentration and separation by Rohde, Ellen, Tomlinson, Andy J, Johnson, Douglas H, Naylor, Stephen

“…Fast and efficient analysis of proteins in physiological fluids is of great interest to researchers and clinicians alike. Capillary electrophoresis (CE) has…”
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Preclinical Efficacy of Bevacizumab with CRLX101, an Investigational Nanoparticle-Drug Conjugate, in Treatment of Metastatic Triple-Negative Breast Cancer by Pham, Elizabeth, Yin, Melissa, Peters, Christian G, Lee, Christina R, Brown, Donna, Xu, Ping, Man, Shan, Jayaraman, Lata, Rohde, Ellen, Chow, Annabelle, Lazarus, Douglas, Eliasof, Scott, Foster, F Stuart, Kerbel, Robert S

“…VEGF pathway-targeting antiangiogenic drugs, such as bevacizumab, when combined with chemotherapy have changed clinical practice for the treatment of a broad…”
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Discovery of a Novel Cabazitaxel Nanoparticle–Drug Conjugate (CRLX522) with Improved Pharmacokinetic Properties and Anticancer Effects Using a β‑Cyclodextrin–PEG Copolymer Based Delivery Platform by Metcalf, Chester A, Svenson, Sonke, Hwang, Jungyeon, Tripathi, Snehlata, Gangal, Geeti, Kabir, Sujan, Lazarus, Douglas, Cole, Roderic, Sweryda-Krawiec, Beata, Shum, Pochi, Brown, Donna, Case, Roy I, van der Poll, Derek, Rohde, Ellen, Harlfinger, Stephanie, Teng, Chi-Hse, Eliasof, Scott

“…Novel nanoparticle–drug conjugates (NDCs) containing diverse, clinically relevant anticancer drug payloads (docetaxel, cabazitaxel, and gemcitabine) were…”
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Abstract 1345: Tumor selective localization of CRLX101, an investigational nanoparticle-drug conjugate of camptothecin by Peters, Christian G., Lazarus, Douglas, Brown, Donna, Zhang, Ningning, Stockmann, Adam P., Case, Roy, Rohde, Ellen, Eliasof, Scott, Jayaraman, Lata

“…Abstract CRLX101, an investigational nanoparticle-drug conjugate (NDC) containing the payload camptothecin, is currently being clinically evaluated in multiple…”
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Abstract 5135: Sustained and controlled in vivo therapeutic levels of drug payloads in tumors using two separate drug combination platforms: Antibody nanoparticle-drug conjugates and multi-drug nanoparticle-drug conjugates, with the potential for improved drug combinability and anticancer effects by Metcalf, Chester A., Poll, Derek van der, Zhao, Liang, Case, Roy I., Lazarus, Doug, Brown, Donna, Halo, Tiffany, Jayaraman, Lata, Peters, Christian, Rohde, Ellen, Eliasof, Scott

“…Abstract Our nanoparticle-drug conjugates (NDCs), created at Cerulean Pharma Inc. by conjugating drug payloads to our novel β-cyclodextrin-PEG (CDP) copolymer,…”
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Potent PDZ-Domain PICK1 Inhibitors that Modulate Amyloid Beta-Mediated Synaptic Dysfunction by Lin, Edward Y. S., Silvian, Laura F., Marcotte, Douglas J., Banos, Charles C., Jow, Flora, Chan, Timothy R., Arduini, Robert M., Qian, Fang, Baker, Darren P., Bergeron, Chris, Hession, Catherine A., Huganir, Richard L., Borenstein, Cassandra F., Enyedy, Istvan, Zou, Jinming, Rohde, Ellen, Wittmann, Marion, Kumaravel, Gnanasambandam, Rhodes, Kenneth J., Scannevin, Robert H., Dunah, Anthone W., Guckian, Kevin M.

“…Protein interacting with C kinase (PICK1) is a scaffolding protein that is present in dendritic spines and interacts with a wide array of proteins through its…”
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Abstract B37: Selective tumor localization of CRLX101, a novel nanoparticle-drug conjugate by Peters, Christian G., Lazarus, Douglas, Brown, Donna, Zhang, Ningning, Case, Roy, Rohde, Ellen, Eliasof, Scott, Jayaraman, Lata

“…Abstract CRLX101, an investigational nanoparticle-drug conjugate (NDC) containing the payload camptothecin conjugated to a biocompatible copolymer of…”
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PRECLINICAL DATA SUPPORTING POTENTIAL EFFICACY OF VERVE-201 - AN INVESTIGATIONAL CRISPR BASE EDITING MEDICINE TARGETING ANGPTL3 - IN PRIMARY HUMAN CELLS, MICE, AND NON-HUMAN PRIMATES by Lee, Richard, Denizio, Jamie, Dutta, Chaitali, Hsu, Hui-Ting, Garrity, Ryan, Pacheco, Alline, Rohde, Ellen, Jayaram, Hari, Kathiresan, Sekar, Bellinger, Andrew, Khera, Amit V.

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Cerebrospinal fluid can be used as a surrogate to assess brain exposures of breast cancer resistance protein and P-glycoprotein substrates by Xiao, Guangqing, Black, Cheryl, Hetu, Gregg, Sands, Eric, Wang, Joy, Caputo, Robin, Rohde, Ellen, Gan, Liang-Shang L

“…The objectives of the study were to characterize the selectivity of dantrolene to breast cancer resistance protein (Bcrp) and to evaluate whether cerebrospinal…”
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Abstract PR10: Significant improvements in therapeutic index for conjugated payloads using a nanoparticle-drug conjugate (NDC) platform to provide sustained drug release and potentially improved anticancer effects by Metcalf, Chester, Poll, Derek van der, Zhao, Liang, Halo, Tiffany, Lazarus, Doug, Stockmann, Adam, Peters, Christian, Brown, Donna, Case, Roy, Rohde, Ellen, Jayaraman, Lata, Wang, Hongwei, Crowell, Tiffany, Senderowicz, Adrian, Eliasof, Scott

“…Abstract Cerulean Pharma Inc.'s Dynamic Tumor Targeting™ Platform creates nanoparticle-drug conjugates (NDCs) designed to significantly mitigate a payload's…”
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Spacer Fidelity Assessments of Guide RNA by Top-Down Mass Spectrometry by Macias, Luis A., Garcia, Sara P., Back, Kayla M., Wu, Yue, Johnson, G. Hall, Kathiresan, Sekar, Bellinger, Andrew M., Rohde, Ellen, Freitas, Michael A., Madsen, James A.

“…The advancement of CRISPR-based gene editing tools into biotherapeutics offers the potential for cures to genetic disorders and for new treatment paradigms for…”
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