Search Results - "Halka, Kathleen G"

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B by Nathwani, Amit C, Reiss, Ulreke M, Tuddenham, Edward G.D, Rosales, Cecilia, Chowdary, Pratima, McIntosh, Jenny, Della Peruta, Marco, Lheriteau, Elsa, Patel, Nishal, Raj, Deepak, Riddell, Anne, Pie, Jun, Rangarajan, Savita, Bevan, David, Recht, Michael, Shen, Yu-Min, Halka, Kathleen G, Basner-Tschakarjan, Etiena, Mingozzi, Federico, High, Katherine A, Allay, James, Kay, Mark A, Ng, Catherine Y.C, Zhou, Junfang, Cancio, Maria, Morton, Christopher L, Gray, John T, Srivastava, Deokumar, Nienhuis, Arthur W, Davidoff, Andrew M

“…In this long-term follow-up study, men with hemophilia B had steady production of functional factor IX after gene transfer with an adeno-associated viral…”
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Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing “Empty Viral Particles” on Safety and Efficacy of Gene Transfer by Nathwani, Amit C, Reiss, Ulrike, Tuddenham, Edward, Chowdary, Pratima, McIntosh, Jenny, Riddell, Anne, Pie, Jun, Mahlangu, Johnny N, Recht, Michael, Shen, Yu-Min, Halka, Kathleen G., Meagher, Michael M, Nienhuis, Arthur W., Davidoff, Andrew M., Mangles, Sarah, Morton, Chris L, Junfang, Zhou, Radulescu, Vlad Calin

“…▪ Background: We have previously shown that a single intravenous administration of a self-complementary adeno-associated virus (scAAV) vector containing a…”
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Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy by Reiss, Ulrike M., Davidoff, Andrew M., Tuddenham, Edward G. D., Chowdary, Pratima, McIntosh, Jenny H., Riddell, Anne, Pie, Arnulfo, Batty, Paul, Calvert, Joanna C. M., Mangles, Sarah, Mahlangu, Johnny, Haley, Kristina M., Recht, Michael, Shen, Yu-Min, Halka, Kathleen G., Zhou, Junfang, Kang, Guolian, Okhomina, Victoria, Morton, Christopher L, Nathwani, Amit C.

“…Background: Previously, we reported data from a first-in-human, dose-escalation, Phase 1 gene therapy trial demonstrating a dose-dependent increase in…”
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Safety and Efficacy of Lower Intensity Induction Therapy with Intravenous Prexigebersen (BP1001) in Patients with High-Risk and Relapsed/Refractory Acute Myeloid Leukemia (AML) by Ohanian, Maro, Lin, Tara L., Ritchie, Ellen, Craig, Michael, Agrawal, Apurv, Halka, Kathleen G., Pemmaraju, Naveen, Borthakur, Gautam, Lim, Miranda, Pierce, Sherry A., Tari Ashizawa, Ana, Kantarjian, Hagop, Cortes, Jorge E., Roboz, Gail J.

“…Background: About two-thirds of AML patients harbor mutations in signaling and kinase pathways (e.g., FLT3, KRAS, NRAS, PTPN11, NF1, KIT), which utilize the…”
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Interim Safety and Efficacy of Lower Intensity Induction Therapy with Intravenous Prexigebersen (BP1001) in Patients with Untreated Acute Myeloid Leukemia (AML) by Ohanian, Maro N., Lin, Tara L, Ritchie, Ellen K., Craig, Michael, Agrawal, Apurv, Halka, Kathleen G., Karur, Vinit, Pemmaraju, Naveen, Borthakur, Gautam, Aminov, Sarah, Lim, Miranda, Pierce, Sherry A., Sacarakis, Natalie, Minor, Stacy, Tari Ashizawa, Ana, Hahne, William, Kantarjian, Hagop M., Cortes, Jorge E., Roboz, Gail J.

“…Background: The growth factor receptor bound protein-2 (Grb2) is vital to tumor progression. Prexigebersen (BP1001) is a liposome-incorporated Grb2 antisense…”
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Melflufen Therapy for Relapsed Refractory Multiple Myeloma (RRMM) Patients Refractory to Daratumumab and/or Pomalidomide: A Report on Early Efficacy by Mateos, Maria-Victoria, Bladé, Joan, Larocca, Alessandra, Oriol, Albert, Rodriguez, Paula, Alegre, Adrián, Cavo, Michele, Moreb, Jan S, Paner, Agne, Gabrail, Nashat, Halka, Kathleen G., Hassoun, Hani, Maisel, Christopher, Mazumder, Amitabha, Zonder, Jeffrey A, Ocio, Enrique M, Byrne, Catriona, Harmenberg, Johan, Thuresson, Sara, Nordstrom, Eva, Lindberg, Jakob, Richardson, Paul G

“…Background: Melflufen is a next generation alkylator, designed for efficient targeting of tumor cells with a unique mechanism of action. Melflufen provides a…”
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Granulopoiesis in long-term culture by marrow from mice with busulfan-induced chronic latent aplasia by Boyd, R L, Caro, J, Halka, K G, Erslev, A J

“…Mice given high-dose busulfan therapy develop a chronic latent marrow aplasia characterized by normal peripheral blood neutrophil numbers, hematocrits and…”
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