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Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Conclusions: Collectively, these data provide strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease. We anticipate that a single dose IV procedure will pose minimal burden to Fabry patients and will be a viable alternative to biweekly enzyme infusions, potentially reducing treatment-related morbidity whislt improving patient quality of life and potentially providing them with a functional long-term cure.DisclosuresKia: Freeline: Employment, Equity Ownership. McIntosh: Freeline: Consultancy. Hosseini: Freeline: Employment, Equit...
Source: Blood - November 21, 2018 Category: Hematology Authors: Kia, A., McIntosh, J., Rosales, C., Hosseini, P., Sheridan, R., Spiewak, J., Mills, K., Corbau, R., Nathwani, A. C. Tags: 801. Gene Therapy and Transfer: Poster I Source Type: research

Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy
This study was supported by the grant of Russian Science Foundation No 16-15-00010. Kazan Federal University was supported by the Russian Government Program of Competitive Growth.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Bashirov, F. V., Salafutdinov, I. I., Sokolov, M. E., Izmailov, A. A., Markosyan, V. A., Fadeev, F. O., Rizvanov, A., Islamov, R. I. Tags: 801. Gene Therapy and Transfer Source Type: research