Zobrazeno 1 - 10
of 93
pro vyhledávání: '"Christian, Brendel"'
Autor:
Denise Klatt, Lucia Sereni, Boya Liu, Pietro Genovese, Axel Schambach, Els Verhoeyen, David A. Williams, Christian Brendel
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102389- (2024)
The baboon endogenous retrovirus (BaEV) glycoprotein is superior to the commonly used vesicular stomatitis virus glycoprotein (VSVg) for retroviral gene transfer into resting hematopoietic stem cells and lymphocyte populations. The derivative BaEVRLe
Externí odkaz:
https://doaj.org/article/8e2e01e0ba9340b482b868e06259719f
Autor:
Daniel C. De Souza, Nicolas Hebert, Erica B. Esrick, M. Felicia Ciuculescu, Natasha M. Archer, Myriam Armant, Étienne Audureau, Christian Brendel, Giuseppe Di Caprio, Frédéric Galactéros, Donghui Liu, Amanda McCabe, Emily Morris, Ethan Schonbrun, Dillon Williams, David K. Wood, David A. Williams, Pablo Bartolucci, John M. Higgins
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-10 (2023)
Abstract We previously reported initial clinical results of post-transcriptional gene silencing of BCL11A expression (NCT 03282656) reversing the fetal to adult hemoglobin switch. A goal of this approach is to increase fetal hemoglobin (HbF) expressi
Externí odkaz:
https://doaj.org/article/c27e3b0ea4ac4641bfd4b6b6b60347e0
Autor:
Jing Zeng, Jerome Ritz, Gabriele Casirati, Andrea Cosentino, Adele Mucci, Mohammed S Mahmoud, Iratxe Ugarte Zabala, Denise Klatt, Christian Brendel, Scott Ficarro, Jarrod Marto, Danilo Pellin, Daniel Bauer, Scott A Armstrong, Pietro Genovese
Publikováno v:
Journal for ImmunoTherapy of Cancer, Vol 11, Iss Suppl 1 (2023)
Externí odkaz:
https://doaj.org/article/83551a902d254e8c92d56c160fced919
Autor:
Hengjiang Ren, Tirth Shah, Hannes Pfeifer, Christian Brendel, Vittorio Peano, Florian Marquardt, Oskar Painter
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-7 (2022)
To realize the full promise of topological mechanical vibrations, they have to be controlled successfully at the nanoscale. Here the authors report topological phonon transport in an optomechanical array. This unlocks the full toolbox of cavity optom
Externí odkaz:
https://doaj.org/article/033c46270bea4cd0967f304ef9f13cd6
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 14-15 (2023)
Externí odkaz:
https://doaj.org/article/539cde975a784ceb980aaa882ac6dd59
Autor:
Alyssa L. Kennedy, Kasiani C. Myers, James Bowman, Christopher J. Gibson, Nicholas D. Camarda, Elissa Furutani, Gwen M. Muscato, Robert H. Klein, Kaitlyn Ballotti, Shanshan Liu, Chad E. Harris, Ashley Galvin, Maggie Malsch, David Dale, John M. Gansner, Taizo A. Nakano, Alison Bertuch, Adrianna Vlachos, Jeffrey M. Lipton, Paul Castillo, James Connelly, Jane Churpek, John R. Edwards, Nobuko Hijiya, Richard H. Ho, Inga Hofmann, James N. Huang, Siobán Keel, Adam Lamble, Bonnie W. Lau, Maxim Norkin, Elliot Stieglitz, Wendy Stock, Kelly Walkovich, Steffen Boettcher, Christian Brendel, Mark D. Fleming, Stella M. Davies, Edie A. Weller, Christopher Bahl, Scott L. Carter, Akiko Shimamura, R. Coleman Lindsley
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-15 (2021)
Understanding the molecular basis of leukaemia predisposition is essential for intervention. The authors here investigate germline genetic leukaemia predisposition by studying Shwachman-Diamond syndrome and report compensatory inactivating mutations
Externí odkaz:
https://doaj.org/article/911d91b30d4548af8f9e72d025592f4b
Autor:
Christian Brendel, Olivier Negre, Michael Rothe, Swaroopa Guda, Geoff Parsons, Chad Harris, Meaghan McGuinness, Daniela Abriss, Alla Tsytsykova, Denise Klatt, Martin Bentler, Danilo Pellin, Lauryn Christiansen, Axel Schambach, John Manis, Helene Trebeden-Negre, Melissa Bonner, Erica Esrick, Gabor Veres, Myriam Armant, David A. Williams
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 589-600 (2020)
In this work we provide preclinical data to support initiation of a first-in-human trial for sickle cell disease (SCD) using an approach that relies on reversal of the developmental fetal-to-adult hemoglobin switch. Erythroid-specific knockdown of BC
Externí odkaz:
https://doaj.org/article/7ebf28bc3d664f328d3cea76508fe211
Autor:
Hee Ho Park, Robinson Triboulet, Martin Bentler, Swaroopa Guda, Peng Du, Haiming Xu, Richard I. Gregory, Christian Brendel, David A. Williams
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 12, Iss , Pp 591-599 (2018)
RNAi-based gene therapy using miRNA-adapted short hairpin RNAs (shRNAmiR) is a powerful approach to modulate gene expression. However, we have observed low viral titers with shRNAmiR-containing recombinant vectors and hypothesized that this could be
Externí odkaz:
https://doaj.org/article/eb62eccf6a0348ee95187fcbf30a46c9
Autor:
Jing Zeng, My Anh Nguyen, Pengpeng Liu, Lucas Ferreira da Silva, Linda Y. Lin, David G. Justus, Karl Petri, Kendell Clement, Shaina N. Porter, Archana Verma, Nola R. Neri, Tolulope Rosanwo, Marioara-Felicia Ciuculescu, Daniela Abriss, Esther Mintzer, Stacy A. Maitland, Selami Demirci, John F. Tisdale, David A. Williams, Lihua Julie Zhu, Shondra M. Pruett-Miller, Luca Pinello, J. Keith Joung, Vikram Pattanayak, John P. Manis, Myriam Armant, Danilo Pellin, Christian Brendel, Scot A. Wolfe, Daniel E. Bauer
Publikováno v:
bioRxiv
SUMMARYGene editing theBCL11Aerythroid enhancer is a validated approach to fetal hemoglobin (HbF) induction for β-hemoglobinopathy therapy, though heterogeneity in edit allele distribution and HbF response may impact its safety and efficacy. Here we
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::250458872ad1c0dd4644117680ac337a
https://doi.org/10.1101/2023.05.27.542323
https://doi.org/10.1101/2023.05.27.542323
Autor:
Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Publikováno v:
Blood. 140:10665-10667
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::e828f889a6fcfac4de9b7fcb45370915
https://doi.org/10.1182/blood-2022-169089
https://doi.org/10.1182/blood-2022-169089