CD14(+)/CD31(+) monocytes expanded by UM171 correct hemophilia A in zebrafish upon lentiviral gene transfer of factor VIII

Emerging gene therapy clinical trials test the correction of hemophilia A (HA) by replacing factor VIII (FVIII) in autologous hematopoietic stem cells (HSCs). Although it is known that platelets, monocyte/macrophages, and mesenchymal stromal cells can secrete transgenic FVIII, a systematic examinati...

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Detalles Bibliográficos
Autores principales: Elnaggar, Muhammad, Al-Mohannadi, Anjud, Hasan, Waseem, Abdelrahman, Doua, Al-Kubaisi, Mohammed J., Pavlovski, Igor, Gentilcore, Giusy, Sathappan, Abbirami, Kizhakayil, Dhanya, Ali, Aesha I., Mohan, Suruchi, Olagunju, Damilola, Cugno, Chiara, Grivel, Jean-Charles, Borsotti, Chiara, Follenzi, Antonia, Da’as, Sahar I., Deola, Sara
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The American Society of Hematology 2022
Materias:
Gene Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9984962/
https://www.ncbi.nlm.nih.gov/pubmed/36477543
http://dx.doi.org/10.1182/bloodadvances.2022009014

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