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Molecular Therapy : the Journal of the American Society of Gene Therapy|November 19, 2025
Base edited skin equivalents with clinical potential for ex vivo correction of recessive dystrophic epidermolysis bullosaAlex Bassons-Bascuñana, Blanca Duarte, Estela Méndez-Jiménez, et al.
The Journal of Pathology|February 10, 2015
BCR-JAK2 drives a myeloproliferative neoplasm in transplanted miceÁlvaro Cuesta-Domínguez, Diego León-Rico, Lara Álvarez, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 12, 2009
Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patientsAriana Jacome, Susana Navarro, Paula Río, et al.
Cell Stem Cell|September 24, 2019
NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi AnemiaFrancisco José Román-Rodríguez, Laura Ugalde, Lara Álvarez, et al.
Nature|June 2, 2009
Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cellsAngel Raya, Ignasi Rodríguez-Pizà, Guillermo Guenechea, et al.
Blood Cancer Journal|August 15, 2021
NKG2D-CAR-transduced natural killer cells efficiently target multiple myelomaAlejandra Leivas, Antonio Valeri, Laura Córdoba, et al.
Molecular Therapy. Oncology|October 27, 2025
Lentiviral-mediated panErbB CAR-T cell therapy against head and neck squamous cell carcinomas for patients with Fanconi anemiaAndrea López, David Charbonnier, Paula Vela, et al.
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