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Human Gene Therapy
|
November 11, 2014
Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade
Sang-oh Han, Songtao Li, Elizabeth D Brooks, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 28, 2026
Imlifidase enables AAV gene therapy in a seropositive Crigler-Najjar patient: On the verge of a paradigm shift
Jeremy Do Cao, Ji Gao-Desliens, Arnaud Valent, et al.
Molecular Therapy. Methods & Clinical Development
|
May 17, 2018
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Zachary Fitzpatrick, Christian Leborgne, Elena Barbon, et al.
Cellular Immunology
|
March 25, 2018
Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients
Christian Leborgne, Virginie Latournerie, Sylvie Boutin, et al.
Chembiochem : a European Journal of Chemical Biology
|
May 19, 2010
Incorporation of 2,3-diaminopropionic acid into linear cationic amphipathic peptides produces pH-sensitive vectors
Yun Lan, Bérangère Langlet-Bertin, Vincenzo Abbate, et al.
Molecular Therapy. Methods & Clinical Development
|
December 25, 2018
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, et al.
Science Advances
|
October 27, 2021
A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia
Volha V Zhukouskaya, Louisa Jauze, Séverine Charles, et al.
Human Molecular Genetics
|
December 20, 2014
Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy
Albert Ribera, Virginia Haurigot, Miguel Garcia, et al.
Molecular Therapy. Methods & Clinical Development
|
October 11, 2016
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, et al.
Scientific Reports
|
January 23, 2020
Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
Berangere Bertin, Philippe Veron, Christian Leborgne, et al.
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of 5
Search research articles
Search
Showing results (31-40 of 47) with videos related to
Sort By:
Page
of 5
Human Gene Therapy
|
November 11, 2014
Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade
Sang-oh Han, Songtao Li, Elizabeth D Brooks, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 28, 2026
Imlifidase enables AAV gene therapy in a seropositive Crigler-Najjar patient: On the verge of a paradigm shift
Jeremy Do Cao, Ji Gao-Desliens, Arnaud Valent, et al.
Molecular Therapy. Methods & Clinical Development
|
May 17, 2018
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Zachary Fitzpatrick, Christian Leborgne, Elena Barbon, et al.
Cellular Immunology
|
March 25, 2018
Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients
Christian Leborgne, Virginie Latournerie, Sylvie Boutin, et al.
Chembiochem : a European Journal of Chemical Biology
|
May 19, 2010
Incorporation of 2,3-diaminopropionic acid into linear cationic amphipathic peptides produces pH-sensitive vectors
Yun Lan, Bérangère Langlet-Bertin, Vincenzo Abbate, et al.
Molecular Therapy. Methods & Clinical Development
|
December 25, 2018
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, et al.
Science Advances
|
October 27, 2021
A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia
Volha V Zhukouskaya, Louisa Jauze, Séverine Charles, et al.
Human Molecular Genetics
|
December 20, 2014
Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy
Albert Ribera, Virginia Haurigot, Miguel Garcia, et al.
Molecular Therapy. Methods & Clinical Development
|
October 11, 2016
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, et al.
Scientific Reports
|
January 23, 2020
Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
Berangere Bertin, Philippe Veron, Christian Leborgne, et al.
Page
of 5