Search Results - "Karlen, Andrea D."

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency by Punwani, Divya, Kawahara, Misako, Yu, Jason, Sanford, Ukina, Roy, Sushmita, Patel, Kiran, Carbonaro, Denise A, Karlen, Andrea D, Khan, Sara, Cornetta, Kenneth, Rothe, Michael, Schambach, Axel, Kohn, Donald B, Malech, Harry L, McIvor, R Scott, Puck, Jennifer M, Cowan, Morton J

“…During B and T lymphocyte maturation, V(D)J recombination is initiated by creation of DNA double-strand breaks. Artemis is an exonuclease essential for their…”
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Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice by Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Erlanson, Olivia, Furcich, Justin, Lund, Troy C., Seelig, Davis, Kitto, Kelley F., Fairbanks, Carolyn A., Kim, Kwi Hye, Buss, Nick, McIvor, R. Scott

“…Mucopolysaccharidosis type II (MPS II) is an X-linked recessive lysosomal disease caused by iduronate-2-sulfatase (IDS) deficiency, leading to accumulation of…”
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Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells by Smith, Miles C, Belur, Lalitha R, Karlen, Andrea D, Erlanson, Olivia, Podetz-Pedersen, Kelly M, McKenzie, Jessica, Detellis, Jenn, Gagnidze, Khatuna, Parsons, Geoffrey, Robinson, Nicholas, Labarre, Shelby, Shah, Saumil, Furcich, Justin, Lund, Troy C, Tsai, Hsing-Chen, McIvor, R Scott, Bonner, Melissa

“…Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is an X-linked recessive lysosomal disease caused by deficiency of iduronate-2-sulfatase (IDS). The…”
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Protection of mice from methotrexate toxicity by ex vivo transduction using lentivirus vectors expressing drug-resistant dihydrofolate reductase by Gori, Jennifer L, Podetz-Pedersen, Kelly, Swanson, Debra, Karlen, Andrea D, Gunther, Roland, Somia, Nikunj V, McIvor, R Scott

“…Methotrexate (MTX) dose-escalation studies were conducted in C57BL/6 mice to determine the chemoprotective effect of transplantation using bone marrow…”
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Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II by Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Podetz-Pedersen, Kelly, Erlanson, Olivia, Laoharawee, Kanut, Furcich, Justin, Lund, Troy C., You, Yun, Seelig, Davis, Webber, Beau R., McIvor, R. Scott

“…Mucopolysaccharidosis type II (Hunter syndrome, MPS II) is an inherited X-linked recessive disease caused by deficiency of iduronate-2-sulfatase (IDS),…”
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Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency by Multhaup, Megan M, Podetz-Pedersen, Kelly M, Karlen, Andrea D, Olson, Erik R, Gunther, Roland, Somia, Nikunj V, Blazar, Bruce R, Cowan, Morton J, McIvor, R Scott

“…Artemis is a single-stranded endonuclease, deficiency of which results in a radiation-sensitive form of severe combined immunodeficiency (SCID-A) most…”
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Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer by Multhaup, Megan, Karlen, Andrea D, Swanson, Debra L, Wilber, Andrew, Somia, Nikunj V, Cowan, Morton J, McIvor, R Scott

“…Artemis is a hairpin-opening endonuclease involved in nonhomologous end-joining and V(D)J recombination. Deficiency of Artemis results in radiation-sensitive…”
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Characterization of the human artemis promoter by heterologous gene expression in vitro and in vivo by Multhaup, Megan M, Gurram, Sweta, Podetz-Pedersen, Kelly M, Karlen, Andrea D, Swanson, Debra L, Somia, Nikunj V, Hackett, Perry B, Cowan, Morton J, McIvor, R Scott

“…Artemis is an endonucleolytic enzyme involved in nonhomologous double-strand break repair and V(D)J recombination. Deficiency of Artemis results in a B- T-…”
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Comparative effectiveness of intravenous and intrathecal AAV9.CB7.hIDS (RGX-121) in a murine model of mucopolysaccharidosis type II by Smith, Miles C., Belur, Lalitha, Erlanson, Olivia L., Furcich, Justin W., Karlen, Andrea D., Lund, Troy C., Buss, Nicholas, McIvor, R. Scott

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Prevention of Cardiac, Neurologic, and Skeletal Manifestations of Murine MPS I by Either Systemic or CSF-Directed AAV9-IDUA/RGX-111 Administered at Minimal Effective Dose by Belur, Lalitha R., Huber, Avery K., Mantone, Hillary, Robertson, Mason, Smith, Miles C., Karlen, Andrea D., Kitto, Kelley F., Ou, Li, Whitley, Chester B., Braunlin, Elizabeth, Furcich, Justin, Lund, Troy C., Seelig, Davis, Fairbanks, Carolyn A., Buss, Nicholas, Kim, Kwi Hye, McIvor, R. Scott

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