Human gene therapy - JoVE Visualize

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Human Gene Therapy|May 21, 2016

Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs DiseaseSubha Karumuthil-Melethil, Sahana Nagabhushan Kalburgi, Patrick Thompson, et al.

Human Gene Therapy|April 3, 2016

Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and FunctionRonald A Bush, Yong Zeng, Peter Colosi, et al.

Human Gene Therapy|May 15, 2016

Advances in Gene Therapy for Diseases of the EyeLolita Petit, Hemant Khanna, Claudio Punzo

Human Gene Therapy|May 15, 2016

Liver-Specific Allergen Gene Transfer by Adeno-Associated Virus Suppresses Allergic Airway Inflammation in MiceCheng-Chi Chan, Chin-Wen Lai, Chia-Jen Wu, et al.

Human Gene Therapy|July 15, 2016

Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced PancreatitisDaniel Gaudet, Erik S Stroes, Julie Méthot, et al.

Human Gene Therapy|August 11, 2017

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine ModelKasun Kodippili, Chady H Hakim, Xiufang Pan, et al.

Human Gene Therapy|August 11, 2017

Designed Ankyrin Repeat Proteins as Her2 Targeting Domains in Chimeric Antigen Receptor-Engineered T CellsElizabeth Siegler, Si Li, Yu Jeong Kim, et al.

Human Gene Therapy|September 19, 2017

Transposons: Moving Forward from Preclinical Studies to Clinical TrialsJaitip Tipanee, Thierry VandenDriessche, Marinee K Chuah

Human Gene Therapy|September 28, 2017

Unbiased Identification of T-Cell Receptors Targeting Immunodominant Peptide-MHC Complexes for T-Cell Receptor ImmunotherapyFelix K M Lorenz, Christian Ellinger, Elisa Kieback, et al.

Human Gene Therapy|September 20, 2017

Comparison of Zinc Finger Nucleases Versus CRISPR-Specific Nucleases for Genome Editing of the Wiskott-Aldrich Syndrome LocusAlejandra Gutierrez-Guerrero, Sabina Sanchez-Hernandez, Giuseppe Galvani, et al.

Pageof 413

Showing results (1051-1060 of 4,124) with videos related to

Sort By:

Pageof 413

Human Gene Therapy|May 21, 2016

Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs DiseaseSubha Karumuthil-Melethil, Sahana Nagabhushan Kalburgi, Patrick Thompson, et al.

Human Gene Therapy|April 3, 2016

Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and FunctionRonald A Bush, Yong Zeng, Peter Colosi, et al.

Human Gene Therapy|May 15, 2016

Advances in Gene Therapy for Diseases of the EyeLolita Petit, Hemant Khanna, Claudio Punzo

Human Gene Therapy|May 15, 2016

Liver-Specific Allergen Gene Transfer by Adeno-Associated Virus Suppresses Allergic Airway Inflammation in MiceCheng-Chi Chan, Chin-Wen Lai, Chia-Jen Wu, et al.

Human Gene Therapy|July 15, 2016

Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced PancreatitisDaniel Gaudet, Erik S Stroes, Julie Méthot, et al.

Human Gene Therapy|August 11, 2017

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine ModelKasun Kodippili, Chady H Hakim, Xiufang Pan, et al.

Human Gene Therapy|August 11, 2017

Designed Ankyrin Repeat Proteins as Her2 Targeting Domains in Chimeric Antigen Receptor-Engineered T CellsElizabeth Siegler, Si Li, Yu Jeong Kim, et al.

Human Gene Therapy|September 19, 2017

Transposons: Moving Forward from Preclinical Studies to Clinical TrialsJaitip Tipanee, Thierry VandenDriessche, Marinee K Chuah

Human Gene Therapy|September 28, 2017

Unbiased Identification of T-Cell Receptors Targeting Immunodominant Peptide-MHC Complexes for T-Cell Receptor ImmunotherapyFelix K M Lorenz, Christian Ellinger, Elisa Kieback, et al.

Human Gene Therapy|September 20, 2017

Comparison of Zinc Finger Nucleases Versus CRISPR-Specific Nucleases for Genome Editing of the Wiskott-Aldrich Syndrome LocusAlejandra Gutierrez-Guerrero, Sabina Sanchez-Hernandez, Giuseppe Galvani, et al.

Pageof 413