Showing results (91-100 of 109) with videos related to

Sort By:
Pageof 11
EMBO Molecular Medicine|July 29, 2010
Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patientsEmanuela Anna Roselli, Riccardo Mezzadra, Marta Claudia Frittoli, et al.
EMBO Molecular Medicine|March 6, 2023
An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse modelMarco Peviani, Sabyasachi Das, Janki Patel, et al.
The Journal of Clinical Investigation|August 3, 2007
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapyAlessandro Aiuti, Barbara Cassani, Grazia Andolfi, et al.
Science Advances|September 12, 2020
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotypeLeslie Weber, Giacomo Frati, Tristan Felix, et al.
Blood|March 10, 2018
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locusChiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 14, 2011
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioningMarshall W Huston, Niek P van Til, Trudi P Visser, et al.
Archives of Disease in Childhood|November 12, 2020
Clinical management of sickle cell liver disease in children and young adultsEirini Kyrana, David Rees, Florence Lacaille, et al.
The Journal of Infectious Diseases|April 6, 2013
Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combinationIsabelle André-Schmutz, Liliane Dal-Cortivo, Emmanuelle Six, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 4, 2024
An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type IISabyasachi Das, Fatlum Rruga, Annita Montepeloso, et al.
Blood|July 19, 2017
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adultEmma C Morris, Thomas Fox, Ronjon Chakraverty, et al.
Pageof 11