Department of Immunology

Andrew M. Scharenberg, M.D.


Andrew M. Scharenberg, MD, is attending physician at Seattle Children’s Hospital, professor in the Department of Pediatrics and adjunct professor in the Department of Immunology at the University of Washington School of Medicine, and a member of the Transplantation Biology Consortium Program at Fred Hutchinson Cancer Research Center. He earned his MD with distinction from the University of North Carolina School of Medicine, performed his residency training in pediatrics at the University of North Carolina Children’s Hospital, and completed a fellowship in immunology at NIH and at the Division of Experimental Pathology, Beth Israel Hospital. He participates in the Seattle Children’s Immunodeficiency Clinic and the Inpatient Immunology Consult Service. At Seattle Children’s Research Institute, he operates a 12-member laboratory that develops technology for targeted genome editing, and co-directs the Program in Cell and Gene Therapy, a program to translate cell and gene therapies for the treatment of inherited immunologic and blood diseases. Dr. Scharenberg is past co-founder of the gene editing pioneer Pregenen Inc, acquired by bluebird bio in 2014. He received the American Pediatric Society/Society for Pediatric Research National Young Investigator Award in 2002; and is an active member of the American Society for Clinical Investigation and the American Pediatric Society.


Department of Pediatrics
University of Washington
Seattle Children’s Research Institute
Box 359300 C9S-6
1900 9th Avenue
Seattle WA 98101
Phone: 206-987-7314
Fax: 206-987-7310


Molecular Immunology
Cancer Immunology







Andrew M. Scharenberg


The Scharenberg laboratory is focused on the development of technology for engineering T-cells and hematopoetic stem cells:

  1. Gene editing methods and technology: Our present efforts are centered on optimizing gene editing efficiency in primary T-cells and hematopoietic stem cells using viral vectors and advanced electroporation methods, and on developing gene edited cell products for hematopoietic genetic diseases and tolerance applications.
  2. Synthetic biology: Next generation cell therapies require new approaches to provide physician control over cell survival, expansion, and potency. We are working on developing orthogonal receptor signaling systems that can be embedded in gene edited cell products to enable precise control over these characteristics.
  3. Vector biology: we are working on developing synthetic vector systems for delivery of gene therapy and gene editing components both ex vivo and in vivo.


  1. High efficiency CRISPR/Cas9-mediated gene editing in primary human T-cells using mutant adenoviral E4orf6/E1b55k “helper” proteins. Gwiazda KS, Grier AE, Sahni J, Burleigh SM, Martin U, Yang JG, Popp NA, Krutein MC, Khan IF, Jacoby K, Jensen MC, Rawlings DJ, Scharenberg AM. Mol Ther. 2016 May 16. doi: 10.1038/mt.2016.105. [Epub ahead of print] PMID: 27203437
  2. Indirect DNA Sequence Recognition and Its Impact on Nuclease Cleavage Activity. Lambert AR, Hallinan JP, Shen BW, Chik JK, Bolduc JM, Kulshina N, Robins LI, Kaiser BK, Jarjour J, Havens K, Scharenberg AM, Stoddard BL. Structure. 2016 Jun 7;24(6):862-73. doi: 10.1016/j.str.2016.03.024. Epub 2016 Apr 28. PMID: 27133026
  3. pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences. Grier AE, Burleigh S, Sahni J, Clough CA, Cardot V, Choe DC, Krutein MC, Rawlings DJ, Jensen MC, Scharenberg AM, Jacoby K. Mol Ther Nucleic Acids. 2016 Apr 19;5:e306. doi: 10.1038/mtna.2016.21. PMID: 27093168
  4. Ethical and regulatory aspects of genome editing. Kohn DB, Porteus MH, Scharenberg AM. Blood. 2016 May 26;127(21):2553-60. doi: 10.1182/blood-2016-01-678136. Epub 2016 Apr 6. Review. PMID: 27053531
  5. Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome. Hubbard N, Hagin D, Sommer K, Song Y, Khan I, Clough C, Ochs HD, Rawlings DJ, Scharenberg AM, Torgerson TR. Blood. 2016 May 26;127(21):2513-22. doi: 10.1182/blood-2015-11-683235. Epub 2016 Feb 22. PMID: 26903548
  6. Digital detection of endonuclease mediated gene disruption in the HIV provirus. Sedlak RH, Liang S, Niyonzima N, De Silva Feelixge HS, Roychoudhury P, Greninger AL, Weber ND, Boissel S, Scharenberg AM, Cheng A, Magaret A, Bumgarner R, Stone D, Jerome KR. Sci Rep. 2016 Feb 2;6:20064. doi: 10.1038/srep20064. PMID: 26829887
  7. Free PMC Article Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases. Osborn MJ, Webber BR, Knipping F, Lonetree CL, Tennis N, DeFeo AP, McElroy AN, Starker CG, Lee C, Merkel S, Lund TC, Kelly-Spratt KS, Jensen MC, Voytas DF, von Kalle C, Schmidt M, Gabriel R, Hippen KL, Miller JS, Scharenberg AM, Tolar J, Blazar BR. Mol Ther. 2016 Mar;24(3):570-81. doi: 10.1038/mt.2015.197. Epub 2015 Oct 27. PMID: 26502778
  8. Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template. Sather BD, Romano Ibarra GS, Sommer K, Curinga G, Hale M, Khan IF, Singh S, Song Y, Gwiazda K, Sahni J, Jarjour J, Astrakhan A, Wagner TA, Scharenberg AM, Rawlings DJ. Sci Transl Med. 2015 Sep 30;7(307):307ra156. doi: 10.1126/scitranslmed.aac5530. PMID: 26424571 Free PMC Article