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The Journal of Clinical Investigation
|
August 3, 2007
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
Alessandro 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 phenotype
Leslie 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 locus
Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 6, 2018
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors
Marta Holstein, Cristina Mesa-Nuñez, Csaba Miskey, 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 conditioning
Marshall 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 adults
Eirini 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 combination
Isabelle André-Schmutz, Liliane Dal-Cortivo, Emmanuelle Six, et al.
Blood
|
July 19, 2017
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult
Emma C Morris, Thomas Fox, Ronjon Chakraverty, et al.
Science Translational Medicine
|
January 24, 2014
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy
Martin K Childers, Romain Joubert, Karine Poulard, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 25, 2011
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy
Annette Deichmann, Martijn H Brugman, Cynthia C Bartholomae, et al.
Page
of 9
Search research articles
Search
Showing results (71-80 of 87) with videos related to
Sort By:
Page
of 9
The Journal of Clinical Investigation
|
August 3, 2007
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
Alessandro 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 phenotype
Leslie 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 locus
Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 6, 2018
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors
Marta Holstein, Cristina Mesa-Nuñez, Csaba Miskey, 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 conditioning
Marshall 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 adults
Eirini 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 combination
Isabelle André-Schmutz, Liliane Dal-Cortivo, Emmanuelle Six, et al.
Blood
|
July 19, 2017
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult
Emma C Morris, Thomas Fox, Ronjon Chakraverty, et al.
Science Translational Medicine
|
January 24, 2014
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy
Martin K Childers, Romain Joubert, Karine Poulard, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 25, 2011
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy
Annette Deichmann, Martijn H Brugman, Cynthia C Bartholomae, et al.
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of 9