Andre Lieber
Medical Genetics
Adjunct Professor

Faculty Information


In 1994, I joined Mark Kay’s newly established Gene Therapy group in Seattle as a postdoctoral fellow of the Germany Academic Exchange Service (DAAD) and German Research Society (DFG). In 1998, I became an independent investigator at the level of Research Assistant Professor in the Division of Medical Genetics. In 2010, I was promoted to full professor, in 2015 “with tenure”. 

In 2010, I co-founded a start-up company, through which we have raised money for two clinical trials with cancer co-therapeutics that were developed in my laboratory.

From 2013 to 2016, I was the Secretary of the American Society of Stem Cell and Gene Therapy, where I was largely responsible for the abstract review process and the poster and oral presentation program. I regularly serve on NIH study sections and I am on the editorial board of 4 gene therapy-related journals.  I am dedicated to motivating students for a medical research career. During the past ten years, I have supervised 17 post-docs, 6 graduate students, 23 undergraduate students, and 5 visiting scientists.

Pronouns: He/Him/His

Education & Training: 
Second Medical Institute
Moscow, Russia
Humboldt University
Berlin, Germany
Elected Member of the American Society of Clinical Investigation
(206) 221-3973
Mailing Address: 

University of Washington

Division of Medical Genetics

1705 NE Pacific St

Box 357720

Seattle, WA 98195-7720

Research & Clinical Interests
Research Interests: 

I am an academic researcher with training in molecular and applied medicine. I studied Medicine and Biochemistry at the Moscow Pirogov Institute and received a PhD at the Humboldt University Berlin. After post-graduate training at the Max Planck Institute for Biochemistry, Berlin, I joined Mark Kay’s lab at the University of Washington as a post-doc in 1994 and became an independent investigator in 1998. In 2015, I was promoted to the rank of Professor of Medicine with Tenure. I have published 127 papers as first or senior author and >50 papers as a co-author. As of 2020, I have supervised 25 post-docs, 7 graduate students, 35 undergraduate students, and 11 visiting scientists.

I have been working on basic and translational aspects of adenoviruses for more than 25 years. I believe that I have made seminal contributions that have advanced the field of basic adenovirus research. Among them is the discovery of two of the known three receptors that are commonly used by human adenoviruses. Two years before the death of Jesse Gelsinger, I have demonstrated that intravenously injected adenovirus vectors triggered critical innate toxicity. My lab was the first to show that adenoviruses interact with blood coagulation factors and that this influences in vivo tropism. My lab has also made contributions to a better understanding of how adenoviruses spread in epithelial tissues.

Examples of translational adenovirus research include new approaches for the treatment of cancer involving recombinant adenovirus proteins. Two of these approaches are currently at the IND stage. The first approach is based on a a small recombinant Ad3 fiber knob derivative (JO) that binds to desmoglein 2 (DSG2), a junction protein that is overexpressed in epithelial cancers. Binding of JO to DSG2 triggers signaling pathways and results in transient opening of tight junctions between tumor cells. This, in turn, increased the intratumoral penetration and efficacy of monoclonal antibodies and chemotherapeutic drugs. JO in combination with PEGylated liposomal doxorubicin will be tested in patients with recurrent ovarian/fallopian tube cancer. The second approach involves a modified recombinant Ad35 fiber knob (Ad35K++). This protein is capable of downregulating CD46, a complement inhibitor, thereby sensitizing leukemia and lymphoma cells to CDC therapy with monocolonal anitbodies. The first clinical trial will be a combination of Ad35K++ and rituximab in patients with CD20+ B-cell maligancies.

In 2016, we published a paper demonstrating the feasibility of a new approach to transduce hematopoietic stem cells (HSCs) in vivo after mobilization. Central to this approach was the idea that mobilized HSCs, transduced with an intravenously injected helper-dependent adenovirus vector (HDAd5/35++) in the peripheral blood circulation, return to the bone marrow and persist there. In the original setting, we used a hyperactive Sleeping Beauty transposase for HDAd5/35++ vector integration and an in vivo selection system to expand transduced HSPCs. Over the last 5 years, we have demonstrated the safety and efficacy of the in vivo gene addition approach in mouse disease models for hemoglobinopathies, hemophilia A, cancer, and infectious diseases. Furthermore, we used non-integrating HDAd5/35++ vectors for genome editing by designer nucleases and, recently, by base and prime editors. We have optimized HSC mobilization as well as prophylactic regiments to prevent innate responses associated with the intravenous injection of HDAd5/35++ vectors. Recent achievements include the cure of Sickle Cell Disease in a mouse model by a single intravenous injection of a therapeutic HDAd5/35++ vector and the demonstration of safety and efficacy of the in vivo approach in five rhesus macaques.

I am a scientific co-founder of a start-up company ( to support the clinical translation of the in vivo HSC gene therapy approach. Our vision is to make HSC gene therapy more cost-efficient and accessible to patients in resource-poor countries.



Shayakhmetov DM, Papayannopoulou T, Stamatoyannopoulos G, Lieber A. (2000) Efficient gene transfer into human CD34+ cells by a retargeted adenovirus vector.  J Virol. 74:2567-83.

Gaggar A, Shayakhmetov DM, Lieber A. (2003) CD46 is a cellular receptor for group B adenovirus. Nat Med, 9: 1408-12.

Li Z, Liu Y, Tuve S, Xun Y, Fan X, Min L, Feng Q, Kiviat N, Kiem HP, Disis ML, Lieber A. (2009) Toward a stem cell gene therapy for breast cancer. Blood. 113(22):5423-33.

Wang H, Liu Y, Li ZY, Liang M, Lieber A. (2009) A recombinant adenovirus type 35 fiber knob protein sensitizes lymphoma cells to rituximab therapy. Blood. 115(3):592-600.

Wang, H, Li ZY, Liu Y, Persson J, Beyer I, Möller T, Koyuncu D, Drescher MR, Strauss R, Zhang XB, Wahl JK, Urban N, Drescher C, Hemminki A, Fender P, Lieber A. (2011) Desmoglein 2 is a receptor for adenovirus serotypes 3, 7, 11, and 14. Nat Med. 17(1):96-104.

Beyer I, van Rensburg R, Strauss R, Li Z, Wang H, Persson J, Yumul R, Feng Q, Song H, Bartek J, Fender P, Lieber A. (2011) Epithelial Junction Opener JO-1 Improves Monoclonal Antibody Therapy of Cancer. Cancer Res. 71(22):7080-90.

Richter M, Yumul R., Wang H, Saydaminova K, Ho M, May D, Baldessari A, Gough M, Drescher C, Urban N, Roffler S, Zubieta C, Carter D, Fender P, Lieber A. (2015) Preclinical safety and efficacy studies with an affinity-enhanced epithelial junction opener and PEGylated liposomal doxorubicin, Molecular Therapeutics: Methods and Clinical Development. 2:15005.

Richter M, Saydaminova K, Yumul R, Krishnan R, Liu J, Nagy EE, Singh M, Izsvák Z, Cattaneo R, Uckert W, Palmer D, Ng P, Haworth KG, Kiem HP, Ehrhardt A, Papayannopoulou T, Lieber A. (2016) In vivo transduction of primitive mobilized hematopoietic stem cells after intravenous injection of integrating adenovirus vectors. Blood. 128:2206-2217.

Wang H, Georgakopoulou A, Psatha N, Li C, Capsali C, Samal HB, Anagnostopoulos A, Ehrhardt A, Izsvak Z, Papayannopoulou T, Lieber A. (2019) In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia. J Clin Invest.129(2):598-615.

Wang H, Liu Z, Li C, Gil S, Papayannopoulou T, Doering CB, Lieber A. (2019) High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells. Blood Adv. 3(19):2883-94.

Li C, Course MM, McNeish IA, Drescher CW, Valdmanis PN, Lieber A. (2020) Prophylactic in vivo hematopoietic stem cell gene therapy with an immune checkpoint inhibitor reverses tumor growth in syngeneic mouse tumor models. Cancer Res. 80(3):549-560.

Wang H, Georgakopoulou A, Li C, Liu Z, Gil S, Bashyam A, Yannaki E, Anagnostopoulos A, Pande A, Izsvák Z, Papayannopoulou T, Lieber A. (2020) Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements. JCI Insights. 5(16):e139538. 

Li C, Wang H, Georgakopoulou A, Gil S, Yannaki E, Lieber A(2020) In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model. Mol Ther. 2020 Sep 5:S1525-0016(20)30458-5.

Li C, Georgakopoulou A, Mishra A, Gil S, Hawkins RD, Yannaki E, Lieber A. (2021) In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal g-globin in β-YAC mice. Blood Adv. 5(4):1122-1135.

Li C, Goncalves KA, Raskó T, Pande A, Gil S, Liu Z, Izcvák Z, Papayannopoulou T, Davis JC, Kiem HP, Lieber A. (2021) Single-dose MGTA-145/plerixafor results in efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice.  Blood Adv. 5(5):1239-124.