Blood Advances | February 23, 2021

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

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Molecular Therapy | September 5, 2020

In vivo HSC gene therapy using a bi-modular HDAd5/35++ vector cures sickle cell disease in a mouse model

Chang Li, Hongjie Wang, Aphrodite Georgakopoulou, Sucheol Gil, Evangelia Yannaki, André Lieber

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JCI Insight | August 20, 2020

Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements

Hongjie Wang, Aphrodite Georgakopoulou, Chang Li, Zhinan Liu, Sucheol Gil, Ashvin Bashyam, Evangelia Yannaki, Achilles Anagnostopoulos, Amit Pande, Zsuzsanna Izsvák, Thalia Papayannopoulou, André Lieber

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Molecular Therapy | December 4, 2019

Targeted integration and high-level transgene expression in AAVS1 transgenic mice after in vivo HSC transduction with HDAd5/35++ vectors

Chang Li, Arpit Suresh Mishra, Sucheol Gil, Meng Wang, Aphrodite Georgakopoulou, Thalia Papayannopoulou, R. David Hawkins, André Lieber

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FEBS Letters | November 20, 2019

Adenovirus vectors in hematopoietic stem cell genome editing

Chang Li and André Lieber

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Blood Advances | October 8, 2019

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

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Science Translational Medicine | July 31, 2019

Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates

Olivier Humbert, Stefan Radtke, Clare Samuelson, Ray R. Carrillo, Anai M. Perez, Sowmya S. Reddy, Christopher Lux, Sowmya Pattabhi, Lauren E. Schefter, Olivier Negre, Ciaran M. Lee, Gang Bao, Jennifer E. Adair, Christopher W. Peterson, David J. Rawlings, Andrew M. Scharenberg, Hans-Peter Kiem

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Stem Cell Reports | June 9, 2019

Autologous, gene-modified hematopoietic stem and progenitor cells repopulate the central nervous system with distinct clonal variants

Christopher W. Peterson, Jennifer E. Adair, Martin E. Wohlfahrt, Claire Deleage, Stefan Radtke, Blake Rust, Krystin K. Norman, Zachary K. Norgaard, Lauren E. Schefter, Gabriella M. Sghia-Hughes, Andrea Repetto, Audrey Baldessari, Robert D. Murnane, Jacob D. Estes, and Hans-Peter Kiem

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The Journal of Clinical Investigation | February 1, 2019

In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

Hongjie Wang, Aphrodite Georgakopoulou, Nikoletta Psatha, Chang Li, Chrysi Capsali, Himanshu Bhusan Samal, Achilles Anagnostopoulos, Anja Ehrhardt, Zsuzsanna Izsvák, Thalia Papayannopoulou, Evangelia Yannaki, André Lieber

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Blood | June 28, 2018

Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing

Chang Li, Nikoletta Psatha, Pavel Sova, Sucheol Gil, Hongjie Wang, Jiho Kim, Chandana Kulkarni, Cristina Valensisi, R. David Hawkins, George Stamatoyannopoulos, André Lieber

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Molecular Therapy Methods & Clinical Development | February 15, 2018

Integrating HDAd5/35++ vectors as a new platform for HSC gene therapy of hemoglobinopathies

Chang Li, Nikoletta Psatha, Hongjie Wang, Manvendra Singh, Himanshu Bhusan Samal, Wenli Zhang, Anja Ehrhardt, Zsuzsanna Izsvá, Thalia Papayannopoulou, André Lieber

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Molecular Therapy Methods & Clinical Development | November 10, 2017

A combined in vivo HSC transduction/selection approach results in efficient and stable gene expression in peripheral blood cells in mice

Hongjie Wang, Maximilian Richter, Nikoletta Psatha, Chang Li, Jiho Kim, Jing Liu, Anja Ehrhardt, Susan K. Nilsson, Benjamin Cao, Donna Palmer, Philip Ng, Zsuzsanna Izsvák, Kevin G. Haworth, Hans-Peter Kiem, Thalia Papayannopoulou, André Lieber

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Science Translational Medicine | November 1, 2017

A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates

Stefan Radtke, Jennifer E. Adair, Morgan A. Giese, Yan-Yi Chan, Zachary K. Norgaard, Mark Enstrom, Kevin G. Haworth, Lauren E. Schefter, and Hans-Peter Kiem

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Blood | November 3, 2016

In vivo transduction of primitive mobilized hematopoietic stem cells after intravenous injection of integrating adenovirus vectors

Maximilian Richter, Kamola Saydaminova, Roma Yumul, Rohini Krishnan, Jing Liu, Eniko-Eva Nagy, Manvendra Singh, Zsuzsanna Izsvák, Roberto Cattaneo, Wolfgang Uckert, Donna Palmer, Philip Ng, Kevin G. Haworth, Hans-Peter Kiem, Anja Ehrhardt, Thalia Papayannopoulou, André Lieber

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The Journal of Clinical Investigation | September 2014

Gene therapy enhances chemotherapy tolerance and efficacy in glioblastoma patients

Jennifer E. Adair, Sandra K. Johnston, Maciej M. Mrugala, Brian C. Beard, Laura A. Guyman, Anne L. Baldock, Carly A. Bridge, Andrea Hawkins-Daarud, Jennifer L. Gori, Donald E. Born, Luis F. Gonzalez-Cuyar, Daniel L. Silbergeld, Russell C. Rockne, Barry E. Storer, Jason K. Rockhill, Kristin R. Swanson, Hans-Peter Kiem

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Cancer Gene Therapy | August 2012

In vivo selection of autologous MGMT gene-modified cells following reduced-intensity conditioning with BCNU and temozolomide in the dog model

J. L. Gori, B. C. Beard, C. Ironside, G. Karponi, Hans-Peter Kiem

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Science Translational Medicine | May 9, 2012

Extended survival of glioblastoma patients after chemoprotective HSC gene therapy

Jennifer E. Adair, Brian C. Beard, Grant D. Trobridge, Tobias Neff, Jason K. Rockhill, Daniel L. Silbergeld, Maciej M. Mrugala, Hans-Peter Kiem

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The Journal of Clinical Investigation | June 14, 2010

Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates

Brian C. Beard, Grant D. Trobridge, Christina Ironside, Jeannine S. McCune, Jennifer E. Adair, Hans-Peter Kiem

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