Zobrazeno 1 - 10
of 90
pro vyhledávání: '"Eduard Ayuso"'
Autor:
Magalie Penaud-Budloo, Emilie Lecomte, Quentin Lecomte, Simon Pacouret, Frédéric Broucque, Aurélien Guy-Duché, Jean-Baptiste Dupont, Laurence Jeanson-Leh, Cécile Robin, Véronique Blouin, Eduard Ayuso, Oumeya Adjali
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 3, Pp 101305- (2024)
With more than 130 clinical trials and 8 approved gene therapy products, adeno-associated virus (AAV) stands as one of the most popular vehicles to deliver therapeutic DNA in vivo. One critical quality attribute analyzed in AAV batches is the presenc
Externí odkaz:
https://doaj.org/article/042640a504cc42909af1d623d410fa67
Autor:
Mathieu Mével, Virginie Pichard, Mohammed Bouzelha, Dimitri Alvarez-Dorta, Pierre-Alban Lalys, Nathalie Provost, Marine Allais, Alexandra Mendes, Elodie Landagaray, Jean-Baptiste Ducloyer, Estelle Toublanc, Anne Galy, Nicole Brument, Gaëlle M. Lefevre, Sébastien G. Gouin, Carolina Isiegas, Guylène Le Meur, Thérèse Cronin, Caroline Le Guiner, Michel Weber, Philippe Moullier, Eduard Ayuso, David Deniaud, Oumeya Adjali
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101187- (2024)
Inherited retinal diseases are a leading and untreatable cause of blindness and are therefore candidate diseases for gene therapy. Recombinant vectors derived from adeno-associated virus (rAAV) are currently the most promising vehicles for in vivo th
Externí odkaz:
https://doaj.org/article/1edecadd12924319b147b4cab1a426f8
Autor:
Aurélien Leray, Pierre-Alban Lalys, Juliette Varin, Mohammed Bouzelha, Audrey Bourdon, Dimitri Alvarez-Dorta, Karine Pavageau, Sébastien Depienne, Maia Marchand, Anthony Mellet, Joanna Demilly, Jean-Baptiste Ducloyer, Tiphaine Girard, Bodvaël Fraysse, Mireille Ledevin, Mickaël Guilbaud, Sébastien G. Gouin, Eduard Ayuso, Oumeya Adjali, Thibaut Larcher, Thérèse Cronin, Caroline Le Guiner, David Deniaud, Mathieu Mével
Publikováno v:
Biomedicine & Pharmacotherapy, Vol 171, Iss , Pp 116148- (2024)
Decades of biological and clinical research have led to important advances in recombinant adeno-associated viruses rAAV-based gene therapy gene therapy. However, several challenges must be overcome to fully exploit the potential of rAAV vectors. Inno
Externí odkaz:
https://doaj.org/article/1ed978775aad41fbbbc3787da57c93b5
Autor:
Michela Milani, Cesare Canepari, Tongyao Liu, Mauro Biffi, Fabio Russo, Tiziana Plati, Rosalia Curto, Susannah Patarroyo-White, Douglas Drager, Ilaria Visigalli, Chiara Brombin, Paola Albertini, Antonia Follenzi, Eduard Ayuso, Christian Mueller, Andrea Annoni, Luigi Naldini, Alessio Cantore
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
“Lentiviral gene therapy to the liver establishes stable long-term normal to supra-normal coagulation factor VIII activity in mouse models of hemophilia A and in non-human primates, representing a potential new treatment option for people with hemo
Externí odkaz:
https://doaj.org/article/c9b97a0d651b468fbb352c8d225f07b9
Autor:
Pauline F. Schmit, Simon Pacouret, Eric Zinn, Elizabeth Telford, Fotini Nicolaou, Frédéric Broucque, Eva Andres-Mateos, Ru Xiao, Magalie Penaud-Budloo, Mohammed Bouzelha, Nicolas Jaulin, Oumeya Adjali, Eduard Ayuso, Luk H. Vandenberghe
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 107-121 (2020)
Generation and screening of libraries of adeno-associated virus (AAV) variants have emerged as a powerful method for identifying novel capsids for gene therapy applications. For the majority of libraries, vast population diversity requires multiplexe
Externí odkaz:
https://doaj.org/article/01e94c81cf474557ad14df40eac9da49
Autor:
Alejandra Gutierrez-Guerrero, Maria Jimena Abrey Recalde, Philippe E. Mangeot, Caroline Costa, Ornellie Bernadin, Séverine Périan, Floriane Fusil, Gisèle Froment, Adriana Martinez-Turtos, Adrien Krug, Francisco Martin, Karim Benabdellah, Emiliano P. Ricci, Simone Giovannozzi, Rik Gijsbers, Eduard Ayuso, François-Loïc Cosset, Els Verhoeyen
Publikováno v:
Frontiers in Genome Editing, Vol 3 (2021)
Programmable nucleases have enabled rapid and accessible genome engineering in eukaryotic cells and living organisms. However, their delivery into human blood cells can be challenging. Here, we have utilized “nanoblades,” a new technology that de
Externí odkaz:
https://doaj.org/article/1d5485f02cd64cd39b1ee599b28883bf
Autor:
Achille François, Mohammed Bouzelha, Emilie Lecomte, Frédéric Broucque, Magalie Penaud-Budloo, Oumeya Adjali, Philippe Moullier, Véronique Blouin, Eduard Ayuso
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 223-236 (2018)
Although the clinical use of recombinant adeno-associated virus (rAAV) vectors is constantly increasing, the development of suitable quality control methods is still needed for accurate vector characterization. Among the quality criteria, the titrati
Externí odkaz:
https://doaj.org/article/e86bc233d9c34383bc042649c09abbea
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 8, Iss C, Pp 166-180 (2018)
Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases. In addition, due to their ability to infect non-dividing and divid
Externí odkaz:
https://doaj.org/article/9c068577e5864e40b1a2db3eeee13e0d
Autor:
Maria Luisa Jaén, Laia Vilà, Ivet Elias, Veronica Jimenez, Jordi Rodó, Luca Maggioni, Rafael Ruiz-de Gopegui, Miguel Garcia, Sergio Muñoz, David Callejas, Eduard Ayuso, Tura Ferré, Iris Grifoll, Anna Andaluz, Jesus Ruberte, Virginia Haurigot, Fatima Bosch
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 6, Iss C, Pp 1-7 (2017)
Diabetes is a complex metabolic disease that exposes patients to the deleterious effects of hyperglycemia on various organs. Achievement of normoglycemia with exogenous insulin treatment requires the use of high doses of hormone, which increases the
Externí odkaz:
https://doaj.org/article/f42af52a68244fbe8b452b2e2e315759
Autor:
Susan D'Costa, Veronique Blouin, Frederic Broucque, Magalie Penaud-Budloo, Achille Fçranois, Irene C Perez, Christine Le Bec, Philippe Moullier, Richard O Snyder, Eduard Ayuso
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Clinical trials using recombinant adeno-associated virus (rAAV) vectors have demonstrated efficacy and a good safety profile. Although the field is advancing quickly, vector analytics and harmonization of dosage units are still a limitation for comme
Externí odkaz:
https://doaj.org/article/abdbfb21a81743f3b6a2cf91a09b3f39