Zobrazeno 1 - 10
of 42
pro vyhledávání: '"Olivier, Negre"'
Autor:
Fabio Ciceri, Régis Peffault de Latour, Raynier Devillier, Patrizia Chiusolo, Aurelie Bauquet, Laura Simons, Pierre Heimendinger, Pierre Gaudeaux, Olivier Negre, Tayebeh-Shabi Soheili, Juliette Paillet, Isabelle Andre, Sebastien Oster, Marina Cavazzana, Frederic Lehmann
Publikováno v:
HemaSphere, Vol 7, p e797368b (2023)
Externí odkaz:
https://doaj.org/article/32f83b6162f34d5f9cea8415c82130c6
Autor:
Jaap Jan Boelens, Muhammad Umair Mushtaq, Joseph Mcguirk, Aurelie Bauquet, Laura Simons, Pierre Heimendinger, Pierre Gaudeaux, Isabelle Andre, Juliette Paillet, Olivier Negre, Tayebeh-Shabi Soheili, Sebastien Oster, Marcel R.M. van den Brink, Frederic Lehmann, Marina Cavazzana, Miguel-Angel Perales
Publikováno v:
HemaSphere, Vol 7, p e660193c (2023)
Externí odkaz:
https://doaj.org/article/6a28ca3eede643b2ac86473747a8877e
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:
Sowmya Pattabhi, Samantha N. Lotti, Mason P. Berger, Swati Singh, Christopher T. Lux, Kyle Jacoby, Calvin Lee, Olivier Negre, Andrew M. Scharenberg, David J. Rawlings
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 17, Iss , Pp 277-288 (2019)
Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit pre
Externí odkaz:
https://doaj.org/article/5c5d0cc4529c44adb9273c8f4a9f26d9
Autor:
Christopher T. Lux, Sowmya Pattabhi, Mason Berger, Cynthia Nourigat, David A. Flowers, Olivier Negre, Olivier Humbert, Julia G. Yang, Calvin Lee, Kyle Jacoby, Irwin Bernstein, Hans-Peter Kiem, Andrew Scharenberg, David J. Rawlings
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 175-183 (2019)
Elements within the γ-hemoglobin promoters (HBG1 and HBG2) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell disease (SCD) subjects with a 13-bp deletion in the HBG1 promoter exhibit a clini
Externí odkaz:
https://doaj.org/article/d1669924b97e44a4beafad9d2ebc6f23
Autor:
Gretchen Lewis, Lauryn Christiansen, Jessica McKenzie, Min Luo, Eli Pasackow, Yegor Smurnyy, Sean Harrington, Philip Gregory, Gabor Veres, Olivier Negre, Melissa Bonner
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss , Pp 313-322 (2018)
Lentiviral vector (LVV)-mediated transduction of human CD34+ hematopoietic stem and progenitor cells (HSPCs) holds tremendous promise for the treatment of monogenic hematological diseases. This approach requires the generation of a sufficient proport
Externí odkaz:
https://doaj.org/article/67f0223fe78441699cfcbacd86785e3a
Autor:
Olivier Negre
Publikováno v:
Cell and Gene Therapy Insights. 7:63-70
Autor:
Olivier Negre, Swaroopa Guda, Christian Brendel, Chad E. Harris, Martin Bentler, Myriam Armant, Melissa Bonner, Erica B. Esrick, John P. Manis, Helene Trebeden-Negre, Axel Schambach, Alla V. Tsytsykova, Danilo Pellin, Michael Rothe, Lauryn Christiansen, Denise Klatt, David A. Williams, Meaghan McGuinness, Daniela Abriss, Geoff Parsons, Gabor Istvan Veres
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 589-600 (2020)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
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
Autor:
David J. Rawlings, Samantha N. Lotti, Olivier Negre, Kyle Jacoby, Swati Singh, Sowmya Pattabhi, Andrew M. Scharenberg, Calvin Lee, Christopher T. Lux, Mason P. Berger
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 17, Iss, Pp 277-288 (2019)
Molecular Therapy. Nucleic Acids
Molecular Therapy. Nucleic Acids
Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit pre
Autor:
Irwin D. Bernstein, Julia G. Yang, Andrew M. Scharenberg, Christopher T. Lux, Olivier Negre, Sowmya Pattabhi, David A. Flowers, Kyle Jacoby, Mason P. Berger, Hans-Peter Kiem, Calvin Lee, Cynthia Nourigat, Olivier Humbert, David J. Rawlings
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss, Pp 175-183 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Elements within the γ-hemoglobin promoters (HBG1 and HBG2) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell disease (SCD) subjects with a 13-bp deletion in the HBG1 promoter exhibit a clini