Ru Xiao, MD, MS

Title: Associate Director, Gene Transfer Vector Core

Educational Background: MD, Sun Yat-Sen University of Medical Sciences; MS, Pharmacology at Sun Yat-Sen University

Research Project: Optimization of the protocol for AAV vector production in large scale; Generation of Eye Disease Models with shRNA approach

Research Interests: Gene therapy in the eye with AAV vectors; AAV vector production in different scale

 Membership: ASGCT

Contact Information: ru_xiao[at]

Lab Phone: 617-573-6990


  1.             Garanto A, Chung DC, Duijkers L, Corral-Serrano JC, Messchaert M, Xiao R, Bennett J, Vandenberghe LH, Collin RW. In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery. Hum Mol Genet. 2016 Apr 22.
  2.             Tabebordbar M. Zhu K, Cheng JK, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science. 2016 Jan 22; 351(6271):407-11

  3.             Zinn E, Pacouret S, Khaychuk V, Turunen HT, Carvalho LS, Andres-Mateos E, Shah S, Shelke R, Maurer AC, Plovie E, Xiao R, Vandenberghe LH. In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector. Cell Rep. 2015 Aug 11;12(6):1056-68

  4.             Vandenberghe LH, Bell P, Maquire AM, Xiao R, Hopkins TB, Grant R, Bennett J, Wilson JM. AAV9 targets cone photoreceptors in the nonhuman primate retina. PloS One. 2013; 8(1): e53463

  5.             Mason JB, Vandenberghe LH, Xiao R, Wilson JM, Richardson DW Influence of serotype, cell type, tissue composition, and time after inoculation on gene expression in recombinant adeno-associated viral vector-transduced equine joint tissues. Am J Vet Res. 2012 Aug;73(8):1178-85

  6.             Vandenberghe LH, Bell P, Maguire AM, Cearley CN, Xiao R, Calcedo R, Wang L, Castle MJ, Maguire AC, Grant R, Wolfe JH, Wilson JM, Bennett J. Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey. Sci Transl Med. 2011 Jun 22;3(88):88ra54.

  7.              Vandenberghe LH, Xiao R, Lock M, Lin J, Korn M, Wilson JM. Efficient serotype- dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. Hum Gene Ther. 2010 Oct;21(10):1251-7.

  8.             Li Y, Lu H, Huang Y, Xiao R, Cai X, He S, Yan G. Glycogen synthase kinases-3beta controls differentiation of malignant glioma cells. Int J Cancer. 2010 Sep 1; 127(6):1271-82.

  9.             Vandenberghe LH, Breous E, Nam HJ, Gao G, Xiao R, Sandhu A, Johnston J, Debyser Z, Agbandje-McKenna M, Wilson JM. Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Ther. 2009 Dec; 16(12):1416-28.

  10.             Wang J, He BJ, Jiang ZB, Zhang YQ, Shan H, Xiao R, Zhang JS, Luo L, Kuang SC, Chen GH, Yang Y. Diagnostic value of multislice spiral CT and MRI in detection of tumor recurrence after liver transplantation for hepatocellular carcinoma. Zhonghua Zhong Liu Za Zhi. 2009 Sep; 31(9):691-6.

  11.             Lauren E.Mays, Luk H. Vandenberghe, Ru Xiao, Peter Bell, Hyun-Joo Nam, Mavis Agbandje-McKenna, and James.M.Wilson. AAV Capsid Structure Drives CD4-Dependent CD8+ T cell Response to Vector Encoded Proteins. J Immunol. 2009 May 15; 182(10):6051-60.

  12.             Zhu W, Ou Y, Li Y, Xiao R, Shu M, Zhou Y, Xie J, He S, Qiu P, Yan G. A Small Molecule Triptolide Suppresses Angiogenesis and Invasion of Human Anaplastic Thyroid Carcinoma Cells via Down-regulation of NF-κB Pathway. Mol Pharmacol. 2009 Apr; 75(4):812-9.

  13.             Zang Linquan, Qiu Pengxin, Xiao R, and Yan Guangmei. Bioinformatics analysis of YZ-2 and preparation of polyclonal antibody. Chin J Cell Mol Immunol 2006, 22(2): 205-7.

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