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Journal Articles

Transduction efficiency and vector biodistribution following prenatal vs neonatal AAV5 gene therapy for Hemophilia A in a sheep model

Martin Rodriguez, Walaa Mohamed, Oluwaseun Babatunde, Bill Vaughan, Andrew Farland, John Owen, Christopher Doering, H. Trent Spencer, Anthony Atala, Christopher Porada, M. Graca Almeida-Porada

Blood (2025) 146 (Supplement 1): 4316.

https://doi.org/10.1182/blood-2025-4316

Published: 2025

Journal Articles

Prenatal transplantation of a clinical cell product engineered to secrete FVIII in a sheep with Hemophilia A corrects the severe bleeding phenotype and prevents hemarthroses

M. Graca Almeida-Porada, Walaa Mohamed, Martin Rodriguez, Oluwaseun Babatunde, Ritu Ramamurthy, Qi Xing, Vladimir Mashanov, Bill Vaughan, Jordan Shields, Andrew Farland, Christopher Doering, H. Trent Spencer, Anthony Atala, Christopher Porada

Blood (2025) 146 (Supplement 1): 1064.

https://doi.org/10.1182/blood-2025-1064

Published: 2025