Selected Publications

Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM: Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9. Mol Ther 29(6): 2019-2029, June 2021.

Wang L, Yang Y, Breton C, Bell P, Li M, Zhang J, Che Y, Saveliev A, He Z, White J, Latshaw C, Xu C, McMenamin D, Yu H, Morizono H, Batshaw ML, Wilson JM.: A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency. Science Advances 6(7): eaax5701, Feburary 2020.

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, June 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, September 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, April 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, March 2016 Notes: (*authors contributed equally).

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), June 2015 Notes: (*authors contributed equally).

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, December 2015.

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, April 2012 Notes: (*authors contributed equally).

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, February 2012.

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, January 2010.

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, November 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, November 2011.

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, March 2007.

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, Aug 2006 Notes: (*authors contributed equally).

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, April 2005.