Search Results - "Merling, Randall K."

Lineage‐specific differentiation of osteogenic progenitors from pluripotent stem cells reveals the FGF1‐RUNX2 association in neural crest‐derived osteoprogenitors by Kidwai, Fahad, Mui, Byron W. H., Arora, Deepika, Iqbal, Kulsum, Hockaday, Madison, Castro Diaz, Luis Fernandez, Cherman, Natasha, Martin, Daniel, Myneni, Vamsee D., Ahmad, Moaz, Futrega, Katarzyna, Ali, Sania, Merling, Randall K., Kaufman, Dan S., Lee, Janice, Robey, Pamela G.

“…Human pluripotent stem cells (hPSCs) can provide a platform to model bone organogenesis and disease. To reflect the developmental process of the human…”
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Molecular Analysis of Neutrophil Differentiation from Human Induced Pluripotent Stem Cells Delineates the Kinetics of Key Regulators of Hematopoiesis by Sweeney, Colin L., Teng, Ruifeng, Wang, Hongmei, Merling, Randall K., Lee, Janet, Choi, Uimook, Koontz, Sherry, Wright, Daniel G., Malech, Harry L.

“…In vitro generation of mature neutrophils from human induced pluripotent stem cells (iPSCs) requires hematopoietic progenitor development followed by myeloid…”
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Transgene-free iPSCs generated from small volume peripheral blood nonmobilized CD34+ cells by Merling, Randall K., Sweeney, Colin L., Choi, Uimook, De Ravin, Suk See, Myers, Timothy G., Otaizo-Carrasquero, Francisco, Pan, Jason, Linton, Gilda, Chen, Lifeng, Koontz, Sherry, Theobald, Narda L., Malech, Harry L.

“…A variety of somatic cells can be reprogrammed to induced pluripotent stem cells (iPSCs), but CD34+ hematopoietic stem cells (HSCs) present in nonmobilized…”
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Neuronal nitric oxide synthase is required for erythropoietin stimulated erythropoiesis in mice by Lee, Jeeyoung, Dey, Soumyadeep, Rajvanshi, Praveen K, Merling, Randall K, Teng, Ruifeng, Rogers, Heather M, Noguchi, Constance T

“…Erythropoietin (EPO), produced in the kidney in a hypoxia responsive manner, is required for red blood cell production. In non-erythroid tissue, EPO increases…”
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Time- and cell-specific activation of BMP signaling restrains chondrocyte hypertrophy by Gadomski, Stephen J., Mui, Byron W.H., Gorodetsky, Raphael, Paravastu, Sriram S., Featherall, Joseph, Li, Li, Haffey, Abigail, Kim, Jae-Chun, Kuznetsov, Sergei A., Futrega, Kathryn, Lazmi-Hailu, Astar, Merling, Randall K., Martin, Daniel, McCaskie, Andrew W., Robey, Pamela G.

“…Stem cell therapies for degenerative cartilage disease are limited by an incomplete understanding of hyaline cartilage formation and maintenance. Human bone…”
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Quantitative Craniofacial Analysis and Generation of Human Induced Pluripotent Stem Cells for Muenke Syndrome: A Case Report by Kidwai, Fahad K, Mui, Byron W H, Almpani, Konstantinia, Jani, Priyam, Keyvanfar, Cyrus, Iqbal, Kulsum, Paravastu, Sriram S, Arora, Deepika, Orzechowski, Pamela, Merling, Randall K, Mallon, Barbara, Myneni, Vamsee D, Ahmad, Moaz, Kruszka, Paul, Muenke, Maximilian, Woodcock, Jeremiah, Gilman, Jeffrey W, Robey, Pamela G, Lee, Janice S

“…In this case report, we focus on Muenke syndrome (MS), a disease caused by the p.Pro250Arg variant in fibroblast growth factor receptor 3 (FGFR3) and…”
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Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction by Sweeney, Colin L., Zou, Jizhong, Choi, Uimook, Merling, Randall K., Liu, Alexander, Bodansky, Aaron, Burkett, Sandra, Kim, Jung-Woong, De Ravin, Suk See, Malech, Harry L.

“…X-linked chronic granulomatous disease (X-CGD) is an immune deficiency resulting from defective production of microbicidal reactive oxygen species (ROS) by…”
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Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease by Merling, Randall K., Kuhns, Douglas B., Sweeney, Colin L., Wu, Xiaolin, Burkett, Sandra, Chu, Jessica, Lee, Janet, Koontz, Sherry, Di Pasquale, Giovanni, Afione, Sandra A., Chiorini, John A., Kang, Elizabeth M., Choi, Uimook, De Ravin, Suk See, Malech, Harry L.

“…Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a…”
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Gene Editing in Chronic Granulomatous Disease by Sweeney, Colin L, Merling, Randall K, De Ravin, Suk See, Choi, Uimook, Malech, Harry L

“…Chronic granulomatous disease (CGD) is an immune deficiency characterized by defects in the production of microbicidal reactive oxygen species (ROS) by the…”
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An AAVS1-Targeted Minigene Platform for Correction of iPSCs From All Five Types of Chronic Granulomatous Disease by Merling, Randall K, Sweeney, Colin L, Chu, Jessica, Bodansky, Aaron, Choi, Uimook, Priel, Debra Long, Kuhns, Douglas B, Wang, Hongmei, Vasilevsky, Sam, De Ravin, Suk See, Winkler, Thomas, Dunbar, Cynthia E, Zou, Jizhong, Zarember, Kol A, Gallin, John I, Holland, Steven M, Malech, Harry L

“…There are five genetic forms of chronic granulomatous disease (CGD), resulting from mutations in any of five subunits of phagocyte oxidase, an enzyme complex…”
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Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers by Hong, So Gun, Yada, Ravi Chandra, Choi, Kyujoo, Carpentier, Arnaud, Liang, T. Jake, Merling, Randall K., Sweeney, Colin L., Malech, Harry L., Jung, Moonjung, Corat, Marcus A.F., AlJanahi, Aisha A., Lin, Yongshun, Liu, Huimin, Tunc, Ilker, Wang, Xujing, Palisoc, Maryknoll, Pittaluga, Stefania, Boehm, Manfred, Winkler, Thomas, Zou, Jizhong, Dunbar, Cynthia E.

“…Nonhuman primate (NHP) induced pluripotent stem cells (iPSCs) offer the opportunity to investigate the safety, feasibility, and efficacy of proposed…”
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Generation of functionally mature neutrophils from induced pluripotent stem cells by Sweeney, Colin L, Merling, Randall K, Choi, Uimook, Priel, Debra Long, Kuhns, Douglas B, Wang, Hongmei, Malech, Harry L

“…Induced pluripotent stem cells (iPSCs) are pluripotent stem cells established from somatic cells. The capability of iPSCs to differentiate into any mature cell…”
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57. Seamless Targeted Correction of CYBB Exon 5 Mutations Restores Granulocyte Function in X-Linked Chronic Granulomatous Disease iPSCs by Sweeney, Colin L., Zou, Jizhong, Choi, Uimook, Merling, Randall K., See DeRavin, Suk, Malech, Harry L.

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557. Targeted CYBB Minigene Insertion into the CYBB Locus for Correction of X-CGD iPSCs Requires Intronic Elements for Expression by Sweeney, Colin L., Choi, Uimook, Zou, Jizhong, Merling, Randall K., Ravin, Suk See De, Malech, Harry L.

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Molecular analysis of neutrophil differentiation from human iPSCs delineates the kinetics of key regulators of hematopoiesis by Sweeney, Colin L., Teng, Ruifeng, Wang, Hongmei, Merling, Randall K., Lee, Janet, Choi, Uimook, Koontz, Sherry, Wright, Daniel G., Malech, Harry L.

“…In vitro generation of mature neutrophils from human induced pluripotent stem cells (iPSCs) requires hematopoietic progenitor development followed by myeloid…”
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332. ZFN-Mediated Minigene or Dinucleotide Gene Correction of p47phox Deficient Autosomal Recessive Chronic Granulomatous Disease iPSC to Generate Oxidase Functional Neutrophils by Merling, Randall K., See De Ravin, Suk, Sweeney, Colin L., Kuhns, Douglas B., Di Pasquale, Giovanni, Chiorini, John A., Malech, Harry L.

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A Platform Minigene AAVS1 Targeted Safe Harbor Approach For Genetic Correction Of iPSC Derived From Patients With Each Of The 5 Genetic Forms Of Chronic Granulomatous Disease by Merling, Randall K, Sweeney, Colin L, Choi, Uimook, Bodansky, Aaron, Chu, Jessica, Priel, Debra Long, Kuhns, Douglas B, Wang, Hongmei, Vasilevsky, Sam, Winkler, Thomas, DeRavin, Suk See, Zou, Jizhong, Leto, Thomas, Dunbar, Cynthia E., Gallin, John, Holland, Steven M., Malech, Harry

“…There are 5 genetic forms of chronic granulomatous disease (CGD), resulting from mutations in one of the CYBB, CYBA, NCF1, NCF2, or NCF4 genes encoding,…”
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Transgene-Free iPSCs Generated from Small Volume Peripheral Blood Non-mobilized CD34+ Cells by Merling, Randall K, Sweeney, Colin L, Choi, Uimook, DeRavin, Suk See, Myers, Timothy G, Otaizo-Carrasquero, Francisco, Pan, Jason, Linton, Gilda, Chen, Lifeng, Koontz, Sherry, Theobald, Narda L, Malech, Harry L

“…A variety of somatic cells can be reprogrammed to induced pluripotent stem cells (iPSCs), but the small number of CD34+ hematopoietic stem cells (HSCs) present…”
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Gene-edited pseudogene resurrection corrects p47 phox -deficient chronic granulomatous disease by Merling, Randall K, Kuhns, Douglas B, Sweeney, Colin L, Wu, Xiaolin, Burkett, Sandra, Chu, Jessica, Lee, Janet, Koontz, Sherry, Di Pasquale, Giovanni, Afione, Sandra A, Chiorini, John A, Kang, Elizabeth M, Choi, Uimook, De Ravin, Suk See, Malech, Harry L

“…Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a…”
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Quantitative Craniofacial Analysis and Generation of Human Induced Pluripotent Stem Cells for Muenke Syndrome: A Case Report by Kidwai, Fahad K, Mui, Byron W H, Almpani, Konstantinia, Jani, Priyam, Keyvanfar, Cyrus, Iqbal, Kulsum, Paravastu, Sriram S, Arora, Deepika, Orzechowski, Pamela, Merling, Randall K, Mallon, Barbara, Myneni, Vamsee D, Ahmad, Moaz, Kruszka, Paul, Muenke, Maximilian, Woodcock, Jeremiah, Gilman, Jeffrey W, Robey, Pamela G, Lee, Janice S

“…In this case report, we focus on Muenke syndrome (MS), a disease caused by the p.Pro250Arg variant in fibroblast growth factor receptor 3 (FGFR3) and…”
Get full text