AAV-mediated genome editing is influenced by the formation of R-loops

Autor: Puzzo, Francesco, Crossley, Magdalena P., Goswami, Aranyak, Zhang, Feijie, Pekrun, Katja, Garzon, Jada L., Cimprich, Karlene A., Kay, Mark A.

Publikováno v: In Molecular Therapy 4 December 2024 32(12):4256-4271

Emotional competence self-help app versus cognitive behavioural self-help app versus self-monitoring app to prevent depression in young adults with elevated risk (ECoWeB PREVENT): an international, multicentre, parallel, open-label, randomised controlled trial

Autor: Watkins, Edward R ^*, Warren, Fiona C, Newbold, Alexandra, Hulme, Claire, Cranston, Timothy, Aas, Benjamin, Bear, Holly, Botella, Cristina, Burkhardt, Felix, Ehring, Thomas, Fazel, Mina, Fontaine, Johnny R J, Frost, Mads, Garcia-Palacios, Azucena, Greimel, Ellen, Hößle, Christiane, Hovasapian, Arpine, Huyghe, Veerle E I, Karpouzis, Kostas, Löchner, Johanna, Molinari, Guadalupe, Pekrun, Reinhard, Platt, Belinda, Rosenkranz, Tabea, Scherer, Klaus R, Schlegel, Katja, Schuller, Bjorn W, Schulte-Korne, Gerd, Suso-Ribera, Carlos, Voigt, Varinka, Voß, Maria, Taylor, Rod S

Publikováno v: In The Lancet Digital Health December 2024 6(12):e894-e903

Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human factor IX variant in mice and rhesus macaques

Autor: Pekrun, Katja, Stephens, Calvin J., Gonzalez-Sandoval, Adriana, Goswami, Aranyak, Zhang, Feijie, Tarantal, Alice F., Blouse, Grant, Kay, Mark A.

Publikováno v: In Molecular Therapy 3 July 2024 32(7):2064-2079

Diagnostic evaluation of paediatric autoimmune lymphoproliferative immunodeficiencies (ALPID): a prospective cohort study

Autor: Abinun, Mario, Abrahamsen, Tore G., Albert, Michael H., Almalky, Mohamed, Altaf, Sadaf, Babayeva, Royala, Bakhtiar, Shahrzad, Baris, Safa, Baumann, Ulrich, Becker, Martina, Beier, Rita, Berger, Thomas, Biebl, Ariane, Bielack, Stefan S., Biskup, Saskia, Bode, Sebastian FN, Borchers, Regine, Boztug, Kaan, Brockmann, Knut, Bruwier, Annelyse, Buchholz, Bernd, Caballero-Oteyza, Andres, Cant, Andrew J., Castro, Carla N., Classen, Carl F., Claviez, Alexander, Crazzolara, Roman, Cuntz, Franziska, Dąbrowska-Leonik, Nel, Derichs, Ute, Dückers, Gregor, Eberl, Wolfgang, Ebetsberger-Dachs, Georg, Erlacher, Miriam, Fabre, Alexandre, Faletti, Laura, Farmand, Susan, Figueiredo, Antonio E., Fischer, Marco, Flaadt, Tim, Full, Hermann, Gambineri, Eleonora, Girschick, Hermann, Goldacker, Sigune, Grimbacher, Bodo, Groß, Miriam, Gruhn, Bernd, Haberfellner, Florian, Hague, Rosie, Hauch, Holger, Hauck, Fabian, Heine, Sabine, Huisman, Elise J., Jakovljevic, Gordana, James, Beki, Janda, Ales, Jones, Neil, Kaiser-Labusch, Petra, Kentouche, Karim, Knight, Julian C., Knirsch, Stephanie, Kontny, Udo, Körholz, Julia, Krenn, Thomas, Kuehnle, Ingrid, Kühne, Thomas, Lee-Dimroth, Jae-Yun, Lehmann, Hartwig, Leipold, Alfred, Meinhardt, Andrea, Minkov, Milen, Mönkemöller, Kirsten, Morbach, Henner, Mücke, Urs, Nathrath, Michaela, Naumann-Bartsch, Nora, Neth, Olaf, Niemeyer, Charlotte M., Olbrich, Peter, Ostró, Róbert, Owens, Stephen, Pac, Malgorzata, Pachlopnik Schmid, Jana, Page, Matthew J.T., Pekrun, Arnulf, Prader, Seraina, Proietti, Michele, Rajacic, Nada, Rothoeft, Tobias, Ryan, Clodagh, Salou, Sarah, Salzer, Elisabeth, Savic, Sinisa, Schilling, Freimut H., Schönberger, Stefan, Schuetz, Catharina, Schuez-Havupalo, Linnea, Schulte, Björn, Schulz, Ansgar, Schuster, Volker, Seidel, Markus G., Siepermann, Kathrin, Skomska-Pawliszak, Malgorzata, Smisek, Petr, Soomann, Maarja, Stiefel, Martina, Storck, Simone, Strahm, Brigitte, Streiter, Monika, Teltschik, Heiko-Manuel, Thalhammer, Julian, Tippelt, Stephan, Toskov, Vasil, Trück, Johannes, Vieth, Simon, von Bismarck, Philipp, Wegehaupt, Oliver, Wiesel, Thomas, Wittkowski, Helmut, Yalcin Gungoren, Ezgi, Hägele, Pauline, Staus, Paulina, Scheible, Raphael, Uhlmann, Annette, Heeg, Maximilian, Klemann, Christian, Maccari, Maria Elena, Ritterbusch, Henrike, Armstrong, Martin, Cutcutache, Ioana, Elliott, Katherine S, von Bernuth, Horst, Leahy, Timothy Ronan, Leyh, Jörg, Holzinger, Dirk, Lehmberg, Kai, Svec, Peter, Masjosthusmann, Katja, Hambleton, Sophie, Jakob, Marcus, Sparber-Sauer, Monika, Kager, Leo, Puzik, Alexander, Wolkewitz, Martin, Lorenz, Myriam Ricarda, Schwarz, Klaus, Speckmann, Carsten, Rensing-Ehl, Anne ^†, Ehl, Stephan ^{†, *}

Publikováno v: In The Lancet Haematology February 2024 11(2):e114-e126

The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner

Autor: Adriana Gonzalez-Sandoval, Katja Pekrun, Shinnosuke Tsuji, Feijie Zhang, King L. Hung, Howard Y. Chang, Mark A. Kay

Publikováno v: Nature Communications, Vol 14, Iss 1, Pp 1-11 (2023)

Abstract Recombinant adeno-associated viral vectors (rAAVs) are among the most commonly used vehicles for in vivo based gene therapies. However, it is hard to predict which AAV capsid will provide the most robust expression in human subjects due to t

Externí odkaz: https://doaj.org/article/d62aaf6b8f6e41b794d5bf3b8b68ef81