<abstract xmlns="http://www.w3.org/1999/xhtml"><p> AAV-mediated gene therapy has the potential to be paradigm shifting as a treatment for haemophilia. A single administration of AAV vector can result in safe and consistent long-term expression of transgene (>5 years), reduction in spontaneous bleeding episodes, reduction in clotting factor usage and an improvement in quality of life. There is huge commercial interest in this approach, with the expectation that an AAV gene therapy product for haemophilia B will be licensed by 2020.</p></abstract>

AAV-mediated gene therapy has the potential to be paradigm shifting as a treatment for haemophilia. A single administration of AAV vector can result in safe and consistent long-term expression of transgene (>5 years), reduction in spontaneous bleeding episodes, reduction in clotting factor usage and an improvement in quality of life. There is huge commercial interest in this approach, with the expectation that an AAV gene therapy product for haemophilia B will be licensed by 2020.

Gene therapy for haemophilia: a very modern success story

The Journal of Haemophilia Practice

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

{"article-title":"Gene therapy for haemophilia: a very modern success story"}

AAV-mediated gene therapy has the potential to be paradigm shifting as a treatment for haemophilia. A single administration of AAV vector can result in safe...

Gene therapy for haemophilia: a very modern success story

Article Category: Kd50: Comprehensive Care

Publicado en línea: 21 abr 2018

Páginas: 26 - 28

DOI: https://doi.org/10.17225/jhp00058

Palabras clave
gene therapy, AAV, haemophilia, Katharine Dormandy

© 2018

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

Gene therapy for haemophilia: a very modern success story

Article Category: Kd50: Comprehensive Care

Publicado en línea: 21 abr 2018

Páginas: 26 - 28

DOI: https://doi.org/10.17225/jhp00058

Palabras clavegene therapy, AAV, haemophilia, Katharine Dormandy

© 2018

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

Palabras clave
gene therapy, AAV, haemophilia, Katharine Dormandy