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Journal Articles

Synthetic Lethality CRISPR Screens in Isogenic iPSCs with Splicing Factor Gene Mutations

Henrik Sperber, PhD, Shailee Vora, BS, MS, Andriana G. Kotini, PhD, Malgorzata Olszewska, MS, Aleksandra Wroblewska, PhD, Brian Brown, PhD, Eirini P. Papapetrou, MD PhD

Blood (2018) 132 (Supplement 1): 3083.

https://doi.org/10.1182/blood-2018-99-111857

Published: 2018

Journal Articles

A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3 + T regulatory cells

Hideto Matsui, Masaru Shibata, Brian Brown, Andrea Labelle, Carol Hegadorn, Chandler Andrews, Marinee Chuah, Thierry VandenDriessche, Carol H. Miao, Christine Hough, David Lillicrap

Blood (2009) 114 (3): 677–685.

https://doi.org/10.1182/blood-2009-03-202267

Published: 2009

Journal Articles

Long-Term Phenotypic Correction of Hemophilia A Mice Following Intravenous Injection of miRNA-Regulated Lentiviral Vectors.

Hideto Matsui, MD, PhD, Margareth Ozelo, MD/PhD, Carol Hegadorn, Andrea Labelle, Erin Burnett, Angie Tuttle, Brian Brown, PhD, Luigi Naldini, MD/PhD, Christine Hough, PhD, David Lillicrap, MD

Blood (2007) 110 (11): 2587.

https://doi.org/10.1182/blood.V110.11.2587.2587

Published: 2007

Journal Articles

Ex Vivo Gene Trasfer for Hemophilia A That Enhances Safety and Results in Sustained In Vivo Factor VIII Expression from Lentivirally-Engineered Endothelial Progenitors.

Hideto Matsui, MD, PhD, Masaru Shibata, MD/PhD, Brian Brown, PhD, Andrea Labelle, Carol Hegadorn, Chandler Andrews, Robert P. Hebbel, MD, Jacques Galipeau, MD, Christine Hough, PhD, David Lillicrap, MD

Blood (2006) 108 (11): 3251.

https://doi.org/10.1182/blood.V108.11.3251.3251

Published: 2006

Journal Articles

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

Marinee K. L. Chuah, Gudrun Schiedner, Lieven Thorrez, Brian Brown, Marion Johnston, Veerle Gillijns, Sabine Hertel, Nico Van Rooijen, David Lillicrap, Désiré Collen, Thierry VandenDriessche, Stefan Kochanek

Blood (2003) 101 (5): 1734–1743.

https://doi.org/10.1182/blood-2002-03-0823

Published: 2003

NARROW

Synthetic Lethality CRISPR Screens in Isogenic iPSCs with Splicing Factor Gene Mutations

A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3 + T regulatory cells

Long-Term Phenotypic Correction of Hemophilia A Mice Following Intravenous Injection of miRNA-Regulated Lentiviral Vectors.

Ex Vivo Gene Trasfer for Hemophilia A That Enhances Safety and Results in Sustained In Vivo Factor VIII Expression from Lentivirally-Engineered Endothelial Progenitors.

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

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ASH Publications

American Society of Hematology

Synthetic Lethality CRISPR Screens in Isogenic iPSCs with Splicing Factor Gene Mutations

A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3 + T regulatory cells

Long-Term Phenotypic Correction of Hemophilia A Mice Following Intravenous Injection of miRNA-Regulated Lentiviral Vectors.

Ex Vivo Gene Trasfer for Hemophilia A That Enhances Safety and Results in Sustained In Vivo Factor VIII Expression from Lentivirally-Engineered Endothelial Progenitors.

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

Email alerts

ASH Publications

American Society of Hematology

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