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The Journal of Clinical Investigation
|
May 3, 2003
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
Federico Mingozzi, Yi-Lin Liu, Eric Dobrzynski, et al.
Human Molecular Genetics
|
April 28, 2018
Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model
Irving Donadon, Mirko Pinotti, Katarzyna Rajkowska, et al.
Human Vaccines & Immunotherapeutics
|
October 26, 2012
Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease
Leslie P Cousens, Federico Mingozzi, Sander van der Marel, et al.
Scientific Reports
|
March 13, 2020
In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery
Cort B Breuer, Killian S Hanlon, Jeya-Shree Natasan, et al.
Blood
|
January 9, 2015
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
Julie M Crudele, Jonathan D Finn, Joshua I Siner, et al.
Molecular Therapy. Methods & Clinical Development
|
August 24, 2019
Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain
Nicola Salvatore Orefice, Benoît Souchet, Jérôme Braudeau, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 23, 2008
Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses
Bernd Hauck, Samuel L Murphy, Peter H Smith, et al.
Blood
|
September 13, 2003
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1
Valder R Arruda, Joerg Schuettrumpf, Roland W Herzog, et al.
Frontiers in Genome Editing
|
April 1, 2022
Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice
Michela Lisjak, Alessia De Caneva, Thibaut Marais, et al.
Human Gene Therapy
|
March 1, 2022
The Effect of Rapamycin and Ibrutinib on Antibody Responses to Adeno-Associated Virus Vector-Mediated Gene Transfer
Zhiquan Xiang, Klaudia Kuranda, William Quinn, et al.
Page
of 14
Search research articles
Search
Showing results (51-60 of 138) with videos related to
Sort By:
Page
of 14
The Journal of Clinical Investigation
|
May 3, 2003
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
Federico Mingozzi, Yi-Lin Liu, Eric Dobrzynski, et al.
Human Molecular Genetics
|
April 28, 2018
Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model
Irving Donadon, Mirko Pinotti, Katarzyna Rajkowska, et al.
Human Vaccines & Immunotherapeutics
|
October 26, 2012
Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease
Leslie P Cousens, Federico Mingozzi, Sander van der Marel, et al.
Scientific Reports
|
March 13, 2020
In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery
Cort B Breuer, Killian S Hanlon, Jeya-Shree Natasan, et al.
Blood
|
January 9, 2015
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
Julie M Crudele, Jonathan D Finn, Joshua I Siner, et al.
Molecular Therapy. Methods & Clinical Development
|
August 24, 2019
Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain
Nicola Salvatore Orefice, Benoît Souchet, Jérôme Braudeau, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 23, 2008
Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses
Bernd Hauck, Samuel L Murphy, Peter H Smith, et al.
Blood
|
September 13, 2003
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1
Valder R Arruda, Joerg Schuettrumpf, Roland W Herzog, et al.
Frontiers in Genome Editing
|
April 1, 2022
Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice
Michela Lisjak, Alessia De Caneva, Thibaut Marais, et al.
Human Gene Therapy
|
March 1, 2022
The Effect of Rapamycin and Ibrutinib on Antibody Responses to Adeno-Associated Virus Vector-Mediated Gene Transfer
Zhiquan Xiang, Klaudia Kuranda, William Quinn, et al.
Page
of 14