Search Results - "THRASHER, A"

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome by Magnani, A., Semeraro, M., Adam, F., Booth, C., Dupré, L., Morris, E. C., Gabrion, A., Roudaut, C., Borgel, D., Toubert, A., Clave, E., Abdo, C., Gorochov, G., Petermann, R., Guiot, M., Miyara, M., Moshous, D., Magrin, E., Denis, A., Suarez, F., Lagresle, C., Roche, A. M., Everett, J., Trinquand, A., Guisset, M., Bayford, J. Xu, Hacein-Bey-Abina, S., Kauskot, A., Elfeky, R., Rivat, C., Abbas, S., Gaspar, H. B., Macintyre, E., Picard, C., Bushman, F. D., Galy, A., Fischer, A., Six, E., Thrasher, A. J., Cavazzana, M.

“…Patients with Wiskott–Aldrich syndrome (WAS) lacking a human leukocyte antigen-matched donor may benefit from gene therapy through the provision of…”
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Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome by Magnani, A., Semeraro, M., Adam, F., Booth, C., Dupré, L., Morris, E. C., Gabrion, A., Roudaut, C., Borgel, D., Toubert, A., Clave, E., Abdo, C., Gorochov, G., Petermann, R., Guiot, M., Miyara, M., Moshous, D., Magrin, E., Denis, A., Suarez, F., Lagresle, C., Roche, A. M., Everett, J., Trinquand, A., Guisset, M., Bayford, J. Xu, Hacein-Bey-Abina, S., Kauskot, A., Elfeky, R., Rivat, C., Abbas, S., Gaspar, H. B., Macintyre, E., Picard, C., Bushman, F. D., Galy, A., Fischer, A., Six, E., Thrasher, A. J., Cavazzana, M.

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The Wiskott-Aldrich syndrome by Ochs, Hans D., Thrasher, Adrian J.

“…The Wiskott-Aldrich syndrome (WAS) is a rare X-linked disorder with variable clinical phenotypes that correlate with the type of mutations in the WAS protein (…”
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The Effects of Coding Schemes on Vibrotactile Biofeedback for Dynamic Balance Training in Parkinson's Disease and Healthy Elderly Individuals by Lee, Beom-Chan, Fung, Alberto, Thrasher, Timothy A.

“…Coding scheme for earlier versions of vibrotactile biofeedback systems for balance-related applications was primarily binary in nature, either on or off at a…”
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Gene silencing dynamics are modulated by transiently active regulatory elements by Vermunt, Marit W., Luan, Jing, Zhang, Zhe, Thrasher, A. Josephine, Huang, Anran, Saari, Megan S., Khandros, Eugene, Beagrie, Robert A., Zhang, Shiping, Vemulamada, Pranay, Brilleman, Matilda, Lee, Kiwon, Yano, Jennifer A., Giardine, Belinda M., Keller, Cheryl A., Hardison, Ross C., Blobel, Gerd A.

“…Transcriptional enhancers have been extensively characterized, but cis-regulatory elements involved in acute gene repression have received less attention…”
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Special Article: Chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient-based registry by Jones, L.B.K.R, McGrogan, P, Flood, T.J, Gennery, A.R, Morton, L, Thrasher, A, Goldblatt, D, Parker, L, Cant, A.J

“…There are no epidemiological studies from the British Isles of chronic granulomatous disease, characterized by recurrent, life-threatening bacterial and fungal…”
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Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients by CHARRIER, S, DUPRE, L, RONCAROLO, M. Grazia, GALY, A, SCARAMUZZA, S, JEANSON-LEH, L, BLUNDELL, M. P, DANOS, O, CATTANEO, F, AIUTI, A, ECKENBERG, R, THRASHER, A. J

“…Gene therapy has been proposed as a potential treatment for Wiskott-Aldrich syndrome (WAS), a severe primary immune deficiency characterized by multiple…”
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A Synchronous Spin-Exchange Optically Pumped NMR-Gyroscope by Sorensen, Susan S., Thrasher, Daniel A., Walker, Thad G.

“…Inertial navigation systems generally consist of timing, acceleration, and orientation measurement units. Although much progress has been made towards…”
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Progress and prospects: gene therapy for inherited immunodeficiencies by Qasim, W, Gaspar, H B, Thrasher, A J

“…Haematopoietic stem cell transplantation (HSCT) is now widely used to treat primary immunodeficiencies (PID). For patients with specific disorders (severe…”
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Translational Mini‐Review Series on Immunodeficiency:
Molecular defects in common variable immunodeficiency by Bacchelli, C., Buckridge, S., Thrasher, A. J., Gaspar, H. B.

“…Summary Common variable immunodeficiency (CVID) is a primary immunodeficiency that typically affects adults and is characterized by abnormalities of quantative…”
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Current progress on gene therapy for primary immunodeficiencies by Zhang, L, Thrasher, A J, Gaspar, H B

“…Primary immunodeficiencies have played a major role in the development of gene therapy for monogenic diseases of the bone marrow. The last decade has seen…”
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Progress and Prospects: Gene Therapy Clinical Trials (Part 1) by ALTON, Eric, BAINBRIDGE, J. W, GREZ, M, THRASHER, A. Z, OTT, M. G, SEGER, R, ALEXANDER, B. L, KAPLITT, M. G, SIMONS, M, YLÄ-HERTTUALA, S, ALL, R. R, GRIESENBACH, U, ALTON, E. W. F. W, FLOTTE, T. R, BRAUN, S, CHENG, S. H, THRASHER, A. J, GASPAR, H. B

“…Over the last two decades gene therapy has moved from preclinical to clinical studies for many diseases ranging from single gene disorders such as cystic…”
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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer by Pike-Overzet, K, Rodijk, M, Ng, Y-Y, Baert, M R M, Lagresle-Peyrou, C, Schambach, A, Zhang, F, Hoeben, R C, Hacein-Bey-Abina, S, Lankester, A C, Bredius, R G M, Driessen, G J A, Thrasher, A J, Baum, C, Cavazzana-Calvo, M, van Dongen, J J M, Staal, F J T

“…Severe combined immunodeficiency (SCID) patients with an inactivating mutation in recombination activation gene 1 ( RAG1 ) lack B and T cells due to the…”
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Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS by Zanta-Boussif, M A, Charrier, S, Brice-Ouzet, A, Martin, S, Opolon, P, Thrasher, A J, Hope, T J, Galy, A

“…The woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) is widely used in retroviral gene transfer vectors. However, this element contains…”
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Single Nucleus Sequencing of Human Colon Myenteric Plexus–Associated Visceral Smooth Muscle Cells, Platelet Derived Growth Factor Receptor Alpha Cells, and Interstitial Cells of Cajal by Schneider, Sabine, Hashmi, Sohaib K., Thrasher, A. Josephine, Kothakapa, Deepika R., Wright, Christina M., Heuckeroth, Robert O.

“…Smooth muscle cells (SMCs), interstitial cells of Cajal (ICCs), and platelet-derived growth factor receptor alpha (PDGFRα+) cells (PαCs) form a functional…”
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Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection by van der Loo, J C M, Swaney, W P, Grassman, E, Terwilliger, A, Higashimoto, T, Schambach, A, Baum, C, Thrasher, A J, Williams, D A, Nordling, D L, Reeves, L, Malik, P

“…The need for γ-retroviral (gRV) vectors with a self-inactivating (SIN) design for clinical application has prompted a shift in methodology of vector…”
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Microbial Characterization during the Early Habitation of the International Space Station by Castro, V. A., Thrasher, A. N., Healy, M., Ott, C. M., Pierson, D. L.

“…An evaluation of the microbiota from air, water, and surface samples provided a baseline of microbial characterization onboard the International Space Station…”
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Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1 by BAINBRIDGE, J. W. B, MISTRY, A, DE ALWIS, M, PALEOLOG, E, BAKER, A, THRASHER, A. J, ALI, R. R

“…Retinal angiogenesis is a central feature of the leading causes of blindness. Current treatments for these conditions are of limited efficacy and cause…”
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Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy by TSCHERNUTTER, M, SCHLICHTENBREDE, F. C, HOWE, S, BALAGGAN, K. S, MUNRO, P. M, BAINBRIDGE, J. W. B, THRASHER, A. J, SMITH, A. J, ALI, R. R

“…The Royal College of Surgeons (RCS) rat is a well-characterized model of autosomal recessive retinitis pigmentosa (RP) due to a defect in the retinal pigment…”
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Neonatal dendritic cells are intrinsically biased against Th‐1 immune responses by LANGRISH, C. L., BUDDLE, J. C., THRASHER, A. J., GOLDBLATT, D.

“…SUMMARY Dendritic cells (DCs) were derived from human peripheral blood monocytes or cord blood monocytes cultured in the presence of IL‐4 and GM‐CSF. Adult and…”
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