Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent

Autor: Camille Lévy, Fouzia Amirache, Anais Girard-Gagnepain, Cecilia Frecha, Francisco J. Roman-Rodríguez, Ornellie Bernadin, Caroline Costa, Didier Nègre, Alejandra Gutierrez-Guerrero, Lenard S. Vranckx, Isabelle Clerc, Naomi Taylor, Lars Thielecke, Kerstin Cornils, Juan A. Bueren, Paula Rio, Rik Gijsbers, François-Loïc Cosset, Els Verhoeyen

Publikováno v: Blood Advances, Vol 1, Iss 23, Pp 2088-2104 (2017)

Abstract: Hematopoietic stem cell (HSC)–based gene therapy trials are now moving toward the use of lentiviral vectors (LVs) with success. However, one challenge in the field remains: efficient transduction of HSCs without compromising their stem ce

Externí odkaz: https://doaj.org/article/d1b4fc861ca4490dae560f0ebb5109ff

GNU Radio based Spectrum Occupancy Measurement Tool

Autor: Marlon Patino, Diana B. Briceno, Francisco J. Roman

Publikováno v: 2022 IEEE International Symposium on Antennas and Propagation and USNC-URSI Radio Science Meeting (AP-S/URSI).

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::c87f4d8f9cd28ba45ee7663d8df6238b
https://doi.org/10.1109/ap-s/usnc-ursi47032.2022.9886076

Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes

Autor: Albert Català, Jesús Fernández, Cristina Díaz-de-Heredia, Raquel Hladun, Jordi Surrallés, Yari Giménez, Ricardo López, Rebeca Sanchez-Dominguez, Lise Larcher, Roser Pujol, Josune Zubicaray, Jonathan D. Schwartz, Nagore García de Andoín, Carmen Azqueta, Elena Sebastián, Francisco J Roman-Rodriguez, Julián Sevilla, R Salgado, José A. Casado, Ana Castillo, Eva M. Galvez, José C. Segovia, Susana Navarro, Sergi Querol, Massimo Bogliolo, Eva Merino, Jean Soulier, Juan A. Bueren, Paula Río, Omaira Alberquilla

Publikováno v: Dipòsit Digital de Documents de la UAB
Universitat Autònoma de Barcelona
Scientia
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss, Pp 66-75 (2021)
Molecular Therapy-Methods & Clinical Development
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname
r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau

Difficulties in the collection of hematopoietic stem and progenitor cells (HSPCs) from Fanconi anemia (FA) patients have limited the gene therapy in this disease. We have investigated (ClinicalTrials.gov, NCT02931071) the safety and efficacy of filgr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3d721cb44cf6570c5a91454504cbfae1
https://ddd.uab.cat/record/249432

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Autor: Massimo Bogliolo, María José Ramírez, Julián Sevilla, Laura Ugalde, Raúl Torres-Ruiz, Juan A. Bueren, Paula Río, Lara Álvarez, Helmut Hanenberg, Jordi Surrallés, Begoña Díez-Cabezas, Francisco J Roman-Rodriguez, Sandra Rodriguez-Perales

Allogeneic hematopoietic stem cell (HSC) transplantation is currently the only curative treatment for the bone marrow failure in Fanconi anemia (FA) patients. However, recent advances in lentiviral-mediated gene therapy have shown that corrected FA H

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6903caf4018c1eb55e23e8d883bf82b6

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

Autor: R Salgado, María L. Lamana, Raquel Hladun, Lara Álvarez, Manfred Schmidt, Laura Cerrato, Julián Sevilla, Jonathan D. Schwartz, Cristina Díaz de Heredia, Juan A. Bueren, Eva Merino, José A. Casado, Paula Río, Anne Galy, M. Luz Lozano, Albert Català, Omaira Alberquilla, Eva M. Galvez, Yari Giménez, Nagore García de Andoín, Anna Raimbault, Rosa Yañez, Roser Pujol, José C. Segovia, Wei Wang, Massimo Bogliolo, Ricardo López, Susana Navarro, Ning Wu, Jordi Barquinero, Guillermo Guenechea, Irina Giralt, Jean Soulier, Jordi Surrallés, Miriam Hernando, Francisco J Roman-Rodriguez, Rebeca Sanchez-Dominguez

Publikováno v: NATURE MEDICINE
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname
r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau
Nature Medicine
Nature Medicine, 2019, 25, pp.1396-1401. ⟨10.1038/s41591-019-0550-z⟩
r-FSJD: Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
Fundació Sant Joan de Déu

Fanconi anemia (FA) is a DNA repair syndrome generated by mutations in any of the 22 FA genes discovered to date1,2. Mutations in FANCA account for more than 60% of FA cases worldwide3,4. Clinically, FA is associated with congenital abnormalities and

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d2b8f8e3a8b6e38248994a628ce12dd6
http://fundanet.fsjd.org/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=16624