Search Results - "Porteus, M."

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice by Barzel, A., Paulk, N. K., Shi, Y., Huang, Y., Chu, K., Zhang, F., Valdmanis, P. N., Spector, L. P., Porteus, M. H., Gaensler, K. M., Kay, M. A.

“…Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus…”
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Interferon priming is essential for human CD34+ cell-derived plasmacytoid dendritic cell maturation and function by Laustsen, A., Bak, R. O., Krapp, C., Kjær, L., Egedahl, J. H., Petersen, C. C., Pillai, S., Tang, H. Q., Uldbjerg, N., Porteus, M., Roan, N. R., Nyegaard, M., Denton, P. W., Jakobsen, M. R.

“…Plasmacytoid dendritic cells (pDC) are essential for immune competence. Here we show that pDC precursor differentiated from human CD34 + hematopoietic stem and…”
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Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs by Ellis, B L, Hirsch, M L, Porter, S N, Samulski, R J, Porteus, M H

“…An emerging strategy for the treatment of monogenic diseases uses genetic engineering to precisely correct the mutation(s) at the genome level. Recent…”
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Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase by Wilkes, M. C., Siva, K., Chen, J., Varetti, G., Youn, M. Y., Chae, H., Ek, F., Olsson, R., Lundbäck, T., Dever, D. P., Nishimura, T., Narla, A., Glader, B., Nakauchi, H., Porteus, M. H., Repellin, C. E., Gazda, H. T., Lin, S., Serrano, M., Flygare, J., Sakamoto, K. M.

“…Diamond Blackfan Anemia (DBA) is a congenital bone marrow failure syndrome associated with ribosomal gene mutations that lead to ribosomal insufficiency. DBA…”
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Treatment decision-making in sickle cell disease patients by Booth, A., Bonham, V., Porteus, M., Ormond, K. E.

“…Sickle cell disease (SCD) is a blood disorder with few treatment options currently available. However, in recent years, there has been much progress toward…”
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CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells by Goodwin, M, Lee, E, Lakshmanan, U, Shipp, S, Froessl, L, Barzaghi, F, Passerini, L, Narula, M, Sheikali, A, Lee, C M, Bao, G, Bauer, C S, Miller, H K, Garcia-Lloret, M, Butte, M J, Bertaina, A, Shah, A, Pavel-Dinu, M, Hendel, A, Porteus, M, Roncarolo, M G, Bacchetta, R

“…The prototypical genetic autoimmune disease is immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, a severe pediatric disease with…”
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Correction to: Treatment decision‑making in sickle cell disease patients by Booth, A., Bonham, V., Porteus, M., Ormond, K. E.

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Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair by Hirsch, M L, Green, L, Porteus, M H, Samulski, R J

“…Adeno-associated virus (AAV) mediates gene targeting in humans by providing exogenous DNA for allelic replacement through homologous recombination. In…”
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Chimeric Nucleases Stimulate Gene Targeting in Human Cells by Porteus, Matthew H., Baltimore, David

“…Correction of gene defects in human somatic cells by targeting as has been used in murine embryonic stem cells has been precluded by the low spontaneous rate…”
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Spatially restricted expression of Dlx-1, Dlx-2 (Tes-1), Gbx-2, and Wnt- 3 in the embryonic day 12.5 mouse forebrain defines potential transverse and longitudinal segmental boundaries by Bulfone, A, Puelles, L, Porteus, MH, Frohman, MA, Martin, GR, Rubenstein, JL

“…The expression patterns of four genes that are potential regulators of development were examined in the CNS of the embryonic day 12.5 mouse embryo. Three of…”
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Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks by Porteus, Matthew H., Cathomen, Toni, Weitzman, Matthew D., Baltimore, David

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Determining age and growth of abalone using stable oxygen isotopes: a tool for fisheries management by Gurney, L.J., Mundy, C., Porteus, M.C.

“…Concerns of inaccuracy or bias associated with current techniques used to obtain age and growth data for abalone (age from shell layers, growth from…”
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Characteristics and Outcome of Children With Beckwith-Wiedemann Syndrome and Wilms’ Tumor: A Report From the National Wilms Tumor Study Group by PORTEUS, M. H, NARKOOL, P, NEUBERG, D, GUTHRIE, K, BRESLOW, N, GREEN, D. M, DILLER, L

“…Children with Beckwith-Wiedemann syndrome (BWS) are at increased risk for developing Wilms' tumor (WT). We reviewed the National Wilms Tumor Study Group…”
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DLX-2, MASH-1, and MAP-2 expression and bromodeoxyuridine incorporation define molecularly distinct cell populations in the embryonic mouse forebrain by Porteus, MH, Bulfone, A, Liu, JK, Puelles, L, Lo, LC, Rubenstein, JL

“…Recently, the Dlx family of homeobox genes have been identified as candidates for regulating patterning and differentiation of the forebrain. We have made a…”
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Isolation and characterization of a novel cDNA clone encoding a homeodomain that is developmentally regulated in the ventral forebrain by Porteus, M H, Bulfone, A, Ciaranello, R D, Rubenstein, J L

“…A complementary DNA, Tes-1, of a novel homeodomain protein has been cloned, and its pattern of expression has been characterized. It is a structural homolog of…”
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Sequence, organization, and transcription of the Dlx-1 and Dlx-2 locus by MCGUINNESS, T, PORTEUS, M. H, SMIGA, S, BULFONE, A, KINGSLEY, C, QIU, M, JEN KUEI LIU, LONG, J. E, XU, D, RUBENSTEIN, J. L. R

“…There are at least five murine Dlx genes that are related to the Drosophila Distal-less homeobox gene. The Dlx genes are primarily expressed in the developing…”
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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus by Antoniani, Chiara, Meneghini, Vasco, Lattanzi, Annalisa, Felix, Tristan, Romano, Oriana, Magrin, Elisa, Weber, Leslie, Pavani, Giulia, El Hoss, Sara, Kurita, Ryo, Nakamura, Yukio, Cradick, Thomas J., Lundberg, Ante S., Porteus, Matthew, Amendola, Mario, El Nemer, Wassim, Cavazzana, Marina, Mavilio, Fulvio, Miccio, Annarita

“…Naturally occurring, large deletions in the β-globin locus result in hereditary persistence of fetal hemoglobin, a condition that mitigates the clinical…”
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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements by Lattanzi, Annalisa, Meneghini, Vasco, Pavani, Giulia, Amor, Fatima, Ramadier, Sophie, Felix, Tristan, Antoniani, Chiara, Masson, Cecile, Alibeu, Olivier, Lee, Ciaran, Porteus, Matthew H., Bao, Gang, Amendola, Mario, Mavilio, Fulvio, Miccio, Annarita

“…Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach for gene therapy of β-thalassemia and sickle cell disease. Using…”
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272 Preclinical development of an autologous stem cell gene therapy platform for treatment of cystic fibrosis sinus and upper airway disease by Bravo, D., Vaidyanathan, S., Baker, J., Tsai, E., Sinha, V., Patel, Z., Hwang, P., Wine, J., Milla, C., Desai, T., Porteus, M., Nayak, J.

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MOF and Histone H4 Acetylation at Lysine 16 Are Critical for DNA Damage Response and Double-Strand Break Repair by Sharma, Girdhar G., So, Sairei, Gupta, Arun, Kumar, Rakesh, Cayrou, Christelle, Avvakumov, Nikita, Bhadra, Utpal, Pandita, Raj K., Porteus, Matthew H., Chen, David J., Cote, Jacques, Pandita, Tej K.

“…Article Usage Stats Services MCB Citing Articles Google Scholar PubMed Related Content Social Bookmarking CiteULike Delicious Digg Facebook Google+ Mendeley…”
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