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Journal Articles

Effective Gene Therapy for Hemophilia a: Novel Re-Dosable Non-Viral Formulation That Provides Stable, and Durable FVIII Expression with Improved Tolerability

Brian Truong, Khal Hajj, Johannes Schwerk, Dongyang Zhang, Johnnatan Tamayo, Gizem Guzelsoy, Joseph Kubicki, Alicia Davis, Lijun Huang, Gopi Vemuri, Kevin Johnson, Dan Smith, Jordan Bendor, Mu Li, Manjunath Goolyam, Daniel Abernathy, Pranav Nyshadham, Fernando Espinoza, Michelle Burrascano, James Yen, Kevin Briseno, Min Tong, Jen Beck, Joseph Lucas, Pavan Battiprolu, Michael Bennett, Blair Madison, Brent Warner, Eric Ostertag, Joshua Rychak
Journal: Blood
Blood (2023) 142 (Supplement 1): 1232.
https://doi.org/10.1182/blood-2023-189784
Published: 2023
Journal Articles

Cas-Clover Editing Efficiency and Off-Target Activity in Human Hepatocytes at the KLKB1 Locus

Oscar Alvarez, Lucas Homa, Stephanie Kearney, Khal Hajj, Zhibo Ma, Cindy Negron, Kevin Briseno, Matt Nitzahn, Nygel Oglesby, Mauricio Barajas-Mora, Arturo Barcenas, Joseph Kubicki, Alicia Davis, Mona Connerney, Pavan Battiprolu, Michael Bennett, Bonnie Jacques, Joshua Rychak, Brent Warner, Blair Madison
Journal: Blood
Blood (2023) 142 (Supplement 1): 7118.
https://doi.org/10.1182/blood-2023-189915
Published: 2023
Journal Articles

Sustained Factor VIII Activity Following Single Dose of Non-Viral Integrating Gene Therapy

Brian Truong, Giang N. Nguyen, Khal Hajj, Joseph Lucas, Johannes Schwerk, James Yen, Kevin Briseno, Michelle Burrascano, Daniel Smith, Johnnatan Tamayo, Fernando Lopez-Espinoza, Michael Bennett, Blair Madison, Denise E. Sabatino, Joshua Rychak
Journal: Blood
Blood (2022) 140 (Supplement 1): 969–970.
https://doi.org/10.1182/blood-2022-167919
Published: 2022