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Lili Wang, PhD

Lili Wang, PhD Researcher

Research Associate Professor of Medicine

Dr. Wang is employed by Penn Medicine.

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Clinical Specialties


American Society of Gene and Cell Therapy (ASGCT), National European Society of Gene and Cell Therapy (ESGCT), International

Hospital Affiliation

Dr. Wang is employed by Penn Medicine.


Description of Research Expertise:

Viral vectors, gene therapy, genome editing, immune response, metabolic diseases and animal models

Selected Publications:

Wang L*, Yang Y*, Breton CA, White J, Zhang J, Che Y, Saveliev A, McMenamin D, He Z, Latshaw C, Li M, Wilson JM (*authors contributed equally): CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice. Blood 133 (26): 2745-2752,2019.

Wang L, Smith J, Breton C, Clark P, Zhang J, Ying L, Che Y, Lape J, Bell P, Calcedo R, Buza EL, Saveliev A, Bartsevich VV, He Z, White J, Li M, Jantz D, Wilson JM: Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol. Nature biotechnology 36 (8): 717-725,2018.

Wang L, Bell P, Morizono H, He Z, Pumbo E, Yu H, White J, Batshaw ML, Wilson JM.: AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice. Mol Genet Metab. 120 (4): 299-305,2017.

Yang Y*, Wang L*, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM.: A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice Nature Biotechnology 34 (3): 334-338,2016.

Wang L, Bell P, Somanathan S, Wang Q, He Z, Yu H, McMenamin D, Goode T, Calcedo R, Wilson JM.: Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids Molecular Therapy 23 (12): 1877-1887,2015.

Bissig-Choisat B*, Wang L*, Legras X, Saha PK, Chen L, Bell P, Pankowicz FP, Hill MC, Barzi M, Leyton CK, Leung HC, Kruse RL, Himes RW, Goss JA, Wilson JM, Chan L, Lagor WR, Bissig KD.: Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model Nature Communications 6 (7339): 2015.

Wang L, Morizono H, Lin J, Bell P, Jones D, McMenamin D, Yu H, Batshaw ML, and Wilson JM.: Preclinical Evaluation of a Clinical Candidate AAV8 Vector for Ornithine Transcarbamylase (OTC) Deficiency Reveals Functional Enzyme from Each Persisting Vector Genome Molecular Genetics and Metabolism 105 (2): 203-211,2012.

Wang L*, Wang H*, Morizono H, Bell P, Jones D, Lin J, McMenamin D, Yu H, Batshaw ML, and Wilson JM. (Authors contributed equally): Sustained Correction of OTC Deficiency in Spf ash Mice Using Optimized Self-complementary AAV2/8 Vectors Gene Therapy 19 (4): 404-10,2012.

Wang L, Louboutin JP, Bell P, Greig J, Li Y, Wu D, and Wilson JM: Muscle-directed Gene Therapy for Hemophilia B with More Efficient and Less Immunogenic AAV Vectors Journal of Thrombosis and Haemostasis 9 (10): 2009-19,2011.

Wang L, Bell P, Lin J, Calcedo R, Tarantal AF, and Wilson JM: AAV8-mediated Hepatic Gene Transfer in Infant Rhesus Monkeys (Macacca mulatta) Molecular Therapy 19 (11): 2012-2020,2011.

Wang L, Calcedo R, Bell P, Lin J, Grant R, Siegel DL, and Wilson JM.: Impact of Pre-existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-associated Virus 8 Vectors Human Gene Therapy 22 (11): 1389-1401,2011.

Wang L, Calcedo R, Wang H, Bell P, Grant R, Vandenberghe LH, Sanmiguel J, Morizono H, Batshaw ML, and Wilson JM: The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques Molecular Therapy 18 (1): 126-134,2010.

Wang L, Figueredo J, Calcedo R, Lin J, and Wilson JM: Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Human Gene Therapy 18 (3): 185-94,2007.

Wang L, Calcedo R, Nichols TC, Bellinger DA, Dillow A, Verma IM, and Wilson JM: Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 105 (8): 3079-86,2005.

Vandenberghe LH*, Wang L*, Somanathan S, Zhi Y, Figueredo J, Calcedo R, Sanmiguel J, Desai RA, Chen CS, Johnston J, Grant RL, Gao G, and Wilson JM: Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nature Medicine 12 (8): 967-71,2006.

Academic Contact Info

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Phone: (215) 746-8929