This is unpublished


Gene Therapy
Human Molecular and Developmental Genetics
Research Assistant Professor, Medical Genetics


Dr. Li earned his PhD in Biochemistry and Molecular Biology from the Wuhan Institute of Virology, Chinese Academy of Sciences in 2015. His PhD projects investigated viral entry mechanisms and established gene therapy approaches for HIV-1 infection. Dr. Li conducted his postdoctoral training at the University of Maryland (2015-2016) and University of Washington (2016-2019). His research focuses on in vivo HSC gene therapy for genetic disorders and infectious diseases. He started his current position as an acting instructor in early 2020.

Education & Training

PhD in Biochemistry and Molecular Biology, Wuhan Institute of Virology, CAS, Wuhan, China (2015)

Research Associate, University of Maryland, College Park, MD (2015-2016)

Senior Fellow, University of Washington, Seattle, WA (2016-2019)


Meritorious Abstract Travel Award (2020)

Meritorious Abstract Travel Award (2019)

Excellence in Research Award (2019)

Outstanding Poster Presentation Award (2018)

Research Interests

My research interests mainly focuses on in vivo HSC gene therapy for beta-hemoglobinopathies adopting various approaches alone or in combination: a) reactivation of fetal hemoglobin by targeting critical motifs in the gamma globin promoter or silencers using genome editing, including precise single nucleotide manipulation, b) gamma-globin gene addition mediated by a hyper-active Sleeping Beauty transposase, and c) targeted globin gene integration into safe genomic loci via homology-directed repair. Combined strategies towards achieving high-level gamma globin expression are being tested in a mouse model of sickle cell disease for complete phenotypic correction and in non-human primates for potential clinical translation. Another more ambitious project is cancer prophylaxis by engineering HSCs in murine models that develop spontaneous cancer. Additionally, I am also interested in developing novel gene therapy approaches for HIV-1 infection based on in vivo transduction of HSCs.



Li C, Lieber A. (2019) Adenovirus vectors in hematopoietic stem cell genome editing. FEBS Lett. 593(24):3623-3648.

Li C, Course MM, McNeish IA, Drescher CW, Valdmanis PN, Lieber A. (2019) Prophylactic in vivo hematopoietic stem cell gene therapy with an immune checkpoint inhibitor reverses tumor growth in a syngeneic mouse tumor model. Cancer Res. 80(3):549-560.

Li C, Mishra AS, Gil S, Wang M, Georgakopoulou A, Papayannopoulou T, Hawkins RD, Lieber A. (2019) Targeted integration and high-level transgene expression in AAVS1 transgenic mice after in vivo HSC transduction with HDAd5/35++ vectors. Mol Ther. 27(12):2195-2212.

Li C, Psatha N, Sova P, Gil S, Wang H, Kim J, Kulkarni C, Valensisi C, Hawkins RD, Stamatoyannopoulos G, Lieber A. (2018) Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing. Blood. 131(26):2915-2928

Li C, Psatha N, Gil S, Wang H, Papayannopoulou T, Lieber A. (2018) HDAd5/35++ adenovirus vector expressing anti-CRISPR peptides decreases CRISPR/Cas9 toxicity in human hematopoietic stem cells. Mol Ther Methods Clin Dev. 9:390-401.

Li C, Psatha N, Wang H, Singh M, Samal HB, Zhang W, Ehrhardt A, Izsvák Z, Papayannopoulou T, Lieber A. (2018) Integrating HDAd5/35++ vectors as a new platform for HSC gene therapy of hemoglobinopathies. Mol Ther Methods Clin Dev. 9:142-152.

Li C, Guan X, Du T, Jin W, Wu B, Liu Y, Wang P, Hu B, Griffin GE, Shattock RJ, Hu Q. (2015) Inhibition of HIV-1 infection of primary CD4+ T-cells by gene editing of CCR5 using adenovirus-delivered CRISPR/Cas9. J Gen Virol. 96(8): 2381-2393.

Li C, Jin W, Du T, Wu B, Liu Y, Shattock RJ, Hu Q. (2014) Binding of HIV-1 virions to alpha4beta 7 expressing cells and impact of antagonizing alpha4beta 7 on HIV-1 infection of primary CD4+ T cells. Virol Sin. 29(6): 381-392.