Product References
Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins.
Nature communications
Pavani G,Laurent M,Fabiano A,Cantelli E,Sakkal A,Corre G,Lenting PJ,Concordet JP,Toueille M,Miccio A,Amendola M
MA1-43012 was used in Western Blotting to describe an ex vivo editing approach to achieve efficient gene targeting in human hematopoietic stem/progenitor cells (HSPCs) and robust expression of clinically relevant proteins by the erythroid lineage.
Wed Jul 29 00:00:00 EDT 2020
Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins.
Nature communications
Pavani G,Laurent M,Fabiano A,Cantelli E,Sakkal A,Corre G,Lenting PJ,Concordet JP,Toueille M,Miccio A,Amendola M
MA1-43012 was used in Western Blotting to describe an ex vivo editing approach to achieve efficient gene targeting in human hematopoietic stem/progenitor cells (HSPCs) and robust expression of clinically relevant proteins by the erythroid lineage.
Wed Jul 29 00:00:00 EDT 2020
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
Molecular therapy. Methods & clinical development
Collaud F,Bortolussi G,Guianvarc'h L,Aronson SJ,Bordet T,Veron P,Charles S,Vidal P,Sola MS,Rundwasser S,Dufour DG,Lacoste F,Luc C,Wittenberghe LV,Martin S,Le Bec C,Bosma PJ,Muro AF,Ronzitti G,Hebben M,Mingozzi F
MA1-43012 was used in an ELISA assay to establish long-term safety and efficiency of gene transder with single strand AAV8-hUGT1A1 in animal models of hemophilia disease.
Fri Mar 15 00:00:00 EDT 2019
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration.
Nature communications
Meliani A,Boisgerault F,Hardet R,Marmier S,Collaud F,Ronzitti G,Leborgne C,Costa Verdera H,Simon Sola M,Charles S,Vignaud A,van Wittenberghe L,Manni G,Christophe O,Fallarino F,Roy C,Michaud A,Ilyinskii P,Kishimoto TK,Mingozzi F
MA1-43012 was used in Enzyme-linked immunosorbent assay to demonstrate that synthetic vaccine particles encapsulating rapamycin, co-administered with adeno-associated virus vectors, prevents the induction of anti-capsid humoral and cell-mediated responses.
Fri Oct 05 00:00:00 EDT 2018
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration.
Nature communications
Meliani A,Boisgerault F,Hardet R,Marmier S,Collaud F,Ronzitti G,Leborgne C,Costa Verdera H,Simon Sola M,Charles S,Vignaud A,van Wittenberghe L,Manni G,Christophe O,Fallarino F,Roy C,Michaud A,Ilyinskii P,Kishimoto TK,Mingozzi F
MA1-43012 was used in Enzyme-linked immunosorbent assay to demonstrate that synthetic vaccine particles encapsulating rapamycin, co-administered with adeno-associated virus vectors, prevents the induction of anti-capsid humoral and cell-mediated responses.
Fri Oct 05 00:00:00 EDT 2018
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.
Molecular therapy. Methods & clinical development
Fitzpatrick Z,Leborgne C,Barbon E,Masat E,Ronzitti G,van Wittenberghe L,Vignaud A,Collaud F,Charles S,Simon Sola M,Jouen F,Boyer O,Mingozzi F
MA1-43012 was used in an ELISA assay to highlight a virtually opposite effect of neutralizing and binding antibodies on AAV vectors transduction.
Fri Jun 15 00:00:00 EDT 2018