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Journal Articles

A simplified, G-CSF-free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model.

Chang Li, Anna Kate Anderson, Peter G Ruminski, Michael P Rettig, Darja Karpova, Hans-Peter Kiem, John F. DiPersio, André Lieber

Blood Adv bloodadvances.2024012757.

https://doi.org/10.1182/bloodadvances.2024012757

Published: 2024

Includes: Supplemental data

Journal Articles

In vivo HSC transduction in rhesus macaques with an HDAd5/3 + vector targeting desmoglein 2 and transiently overexpressing cxcr4

Hongjie Wang, Audrey Germond, Chang Li, Sucheol Gil, Jiho Kim, Hans-Peter Kiem, André Lieber

Blood Adv (2022) 6 (15): 4360–4372.

https://doi.org/10.1182/bloodadvances.2022007975

Published: 2022

Includes: Supplemental data

Journal Articles

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

Chang Li, Kevin A. Goncalves, Tamás Raskó, Amit Pande, Sucheol Gil, Zhinan Liu, Zsuzsanna Izsvák, Thalia Papayannopoulou, John C. Davis, Jr, Hans-Peter Kiem, André Lieber

Blood Adv (2021) 5 (5): 1239–1249.

https://doi.org/10.1182/bloodadvances.2020003714

Published: 2021

Includes: Supplemental data

Journal Articles

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

Chang Li, Aphrodite Georgakopoulou, Arpit Mishra, Sucheol Gil, R. David Hawkins, Evangelia Yannaki, André Lieber

Blood Adv (2021) 5 (4): 1122–1135.

https://doi.org/10.1182/bloodadvances.2020003702

Published: 2021

Includes: Supplemental data

Journal Articles

High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

Hongjie Wang, Zhinan Liu, Chang Li, Sucheol Gil, Thalia Papayannopoulou, Christopher B. Doering, André Lieber

Blood Adv (2019) 3 (19): 2883–2894.

https://doi.org/10.1182/bloodadvances.2019000706

Published: 2019

Includes: Supplemental data

NARROW

A simplified, G-CSF-free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model.

In vivo HSC transduction in rhesus macaques with an HDAd5/3 + vector targeting desmoglein 2 and transiently overexpressing cxcr4

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

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ASH Publications

American Society of Hematology

A simplified, G-CSF-free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model.

In vivo HSC transduction in rhesus macaques with an HDAd5/3 + vector targeting desmoglein 2 and transiently overexpressing cxcr4

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

Email alerts

ASH Publications

American Society of Hematology

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