Search Results - "BATTY, Paul"

Advances and challenges for hemophilia gene therapy by Batty, Paul, Lillicrap, David

“…Abstract Hemophilia is an X-linked inherited bleeding disorder, resulting from defects in the F8 (hemophilia A) or F9 (hemophilia B) genes. Persons with…”
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Hemophilia Gene Therapy: Approaching the First Licensed Product by Batty, Paul, Lillicrap, David

“…The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be…”
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A mitotic chromatin phase transition prevents perforation by microtubules by Schneider, Maximilian W. G., Gibson, Bryan A., Otsuka, Shotaro, Spicer, Maximilian F. D., Petrovic, Mina, Blaukopf, Claudia, Langer, Christoph C. H., Batty, Paul, Nagaraju, Thejaswi, Doolittle, Lynda K., Rosen, Michael K., Gerlich, Daniel W.

“…Dividing eukaryotic cells package extremely long chromosomal DNA molecules into discrete bodies to enable microtubule-mediated transport of one genome copy to…”
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Rituximab, used alone or in combination, is superior to other treatment modalities in splenic marginal zone lymphoma by Else, Monica, Marín-Niebla, Ana, de la Cruz, Fátima, Batty, Paul, Ríos, Eduardo, Dearden, Claire E., Catovsky, Daniel, Matutes, Estella

“…Summary Splenic marginal zone lymphoma (SMZL) is a rare B‐cell malignancy, with no standard treatment other than splenectomy. Rituximab has shown encouraging…”
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Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action by Liu, Su, Razon, Lisa, Ritchie, Olivia, Sihn, Choong-Ryoul, Handyside, Britta, Berguig, Geoffrey, Woloszynek, Jill, Zhang, Lening, Batty, Paul, Lillicrap, David, Agrawal, Vishal, Cortesio, Christa, Gebretsadik, Kahsay, Akeefe, Hassibullah, Colosi, Peter, Kim, Benjamin, Bunting, Stuart, Fong, Sylvia

“…Recombinant adeno-associated virus (AAV) is an effective platform for therapeutic gene transfer; however, tissue-tropism differences between species are a…”
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Adeno-associated viral vector integration: implications for long-term efficacy and safety by Batty, Paul, Lillicrap, David

“…Adeno-associated virus (AAV) vector gene therapy provides a promising platform for treatment of monogenic inherited disorders. Clinical studies have…”
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Gene therapy for hemophilia: Current status and laboratory consequences by Batty, Paul, Lillicrap, David

“…Since the cloning and characterization of the factor VIII (FVIII) and factor IX genes in the mid‐1980s, gene therapy has been perceived as having significant…”
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Mitotic Chromosome Mechanics: How Cells Segregate Their Genome by Batty, Paul, Gerlich, Daniel W.

“…During mitosis, replicated chromosomes segregate such that each daughter cell receives one copy of the genome. Faithful mechanical transport during mitosis…”
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Advances in knowledge of inhibitor formation in severe haemophilia A by Cormier, Matthew, Batty, Paul, Tarrant, Julie, Lillicrap, David

“…Summary Anti‐drug antibody formation following factor VIII (FVIII) replacement therapy is the most important treatment‐related complication in patients with…”
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Transcriptomic Profiling to Understand Inhibitor Development in Previously Untreated Patients with Severe Hemophilia a by Batty, Paul, Watson, David, Wozniak, Eva, Mein, Charles, Barnes, Michael

“…Background: When starting treatment with factor VIII (FVIII) concentrate in individuals with severe hemophilia A, minimizing the risk of FVIII inhibitor…”
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Sustainable urban rail systems: Strategies and technologies for optimal management of regenerative braking energy by González-Gil, Arturo, Palacin, Roberto, Batty, Paul

“…•Review of principal regenerative braking strategies and technologies for urban rail.•Different energy storage technologies are assessed for use in urban…”
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Childhood Treatment with Adeno-Associated Viral Gene Therapy Results in Stable FVIII Expression and Improved Bleeding Phenotype in Adult Severe Hemophilia A Dogs by Batty, Paul, Menard, Alex, Mo, Aomei, Brown, Christine, Ismail, Ashrafali M, Yates, Bridget, Swystun, Laura, Harpell, Lori, Pender, Abbey, Winterborn, Andrew, Fong, Sylvia, Lillicrap, David

“…Introduction: In patients with severe hemophilia A, adeno-associated virus (AAV)-mediated factor VIII (FVIII) gene therapy is associated with reduced bleeding…”
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Vector integration and fate in the hemophilia dog liver multiple years after AAV-FVIII gene transfer by Batty, Paul, Fong, Sylvia, Franco, Matteo, Sihn, Choong-Ryoul, Swystun, Laura L., Afzal, Saira, Harpell, Lorianne, Hurlbut, David, Pender, Abbey, Su, Cheng, Thomsen, Hauke, Wilson, Christopher, Youssar, Loubna, Winterborn, Andrew, Gil-Farina, Irene, Lillicrap, David

“…•In hemophilia A dogs, AAV-cFVIII vectors predominantly persist in the liver long-term in nonintegrated episomal forms.•AAV vector integration was seen at low…”
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Cohesin mediates DNA loop extrusion and sister chromatid cohesion by distinct mechanisms by Nagasaka, Kota, Davidson, Iain F, Stocsits, Roman R, Tang, Wen, Wutz, Gordana, Batty, Paul, Panarotto, Melanie, Litos, Gabriele, Schleiffer, Alexander, Gerlich, Daniel W, Peters, Jan-Michael

“…Cohesin connects CTCF-binding sites and other genomic loci in cis to form chromatin loops and replicated DNA molecules in trans to mediate sister chromatid…”
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Cohesin‐mediated DNA loop extrusion resolves sister chromatids in G2 phase by Batty, Paul, Langer, Christoph CH, Takács, Zsuzsanna, Tang, Wen, Blaukopf, Claudia, Peters, Jan‐Michael, Gerlich, Daniel W

“…Genetic information is stored in linear DNA molecules, which are highly folded inside cells. DNA replication along the folded template path yields two sister…”
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Postpartum bleeding in women with inherited bleeding disorders: a matched cohort study by Wolf, Sonia, Sardo Infirri, Sofia, Batty, Paul, Sahar, Bazgha, Beski, Shoreh, Bowles, Louise

“…Women with inherited bleeding disorders (IBDs) are reported to have higher rates of primary and secondary postpartum haemorrhage (PPH), even with optimal…”
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Releasing Activity Disengages Cohesin’s Smc3/Scc1 Interface in a Process Blocked by Acetylation by Beckouët, Frederic, Srinivasan, Madhusudhan, Roig, Maurici Brunet, Chan, Kok-Lung, Scheinost, Johanna C., Batty, Paul, Hu, Bin, Petela, Naomi, Gligoris, Thomas, Smith, Alexandra C., Strmecki, Lana, Rowland, Benjamin D., Nasmyth, Kim

“…Sister chromatid cohesion conferred by entrapment of sister DNAs within a tripartite ring formed between cohesin’s Scc1, Smc1, and Smc3 subunits is created…”
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UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults by Chowdary, Pratima, Duran, Beatriz, Batty, Paul, Lowe, Gillian, Jones, April, Pollard, Debra, Boyce, Sara, Motwani, Jayashree, Amirloo, Bahareh, Musgrave, Kathryn, Hopper, David, Classey, Stephen, Whitaker, Sarah, Dunn, Nicola, Bowyer, Annette, Shapiro, Susan

“…2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons…”
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Vector integration and fate in the hemophilia dog liver multi-years following AAV-FVIII gene transfer by Batty, Paul, Fong, Sylvia, Franco, Matteo, Sihn, Choong-Ryoul, Swystun, Laura L, Afzal, Saira, Harpell, Lori M, Hurlbut, David, Pender, Abbey, Su, Cheng, Thomsen, Ph.D, Hauke, Wilson, Christopher, Youssar, Loubna, Winterborn, Andrew, Gil-Farina, Irene, Lillicrap, David

“…Gene therapy using adeno-associated viral (AAV) vectors is a promising approach for the treatment of monogenic disorders. Long-term multi-year transgene…”
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GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant by Chowdary, Pratima, Reiss, Ulrike M., Tuddenham, Edward G. D., Batty, Paul, McIntosh, Jenny H., Radulescu, Vlad Calin, Chang, Eugenia, Laffan, Michael A., Riddell, Anne, Calvert, Joanna C. M., Pie, Arnulfo, Peralta, Rita, Dissanayake, Upuli Pabakumari, Kamel, Kirollos, Sreedhar, Bharath Ram, Zhou, Junfang, Kang, Guolian, Olufadi, Yunus, Meagher, Michael M, Morton, Christopher L, Evans, Alison J., Davidoff, Andrew M., Nathwani, Amit C.

“…Background: GO-8 (ClinicalTrials.gov: NCT03001830) is a study of liver-directed adeno-associated virus (AAV) gene therapy for haemophilia A (HA) that uses a…”
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