Salk Institute
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Leszek  Lisowski

Leszek Lisowski

Gene Transfer, Targeting and Therapeutics Core

As Director of the Gene Transfer, Targeting and Therapeutics Facility at the Salk Institute, Leszek Lisowski's main focus is on providing comprehensive resources to facilitate the safe and effective use of viral and non-viral gene transfer technologies to study critical problems in all aspects of biological research.

Dr. Lisowski has extensive background in gene delivery and targeting using multiple viral-vector systems. During his Ph.D. training at Cornell University, he developed new and improved lentiviral vectors (LV) for the treatment of beta-thalassemia and hemophilia. He also performed extensive work on the assessment of vector toxicity and the risk of insertional oncogenesis. These studies led to his long-standing interest in viral vectors as vehicles for gene replacement therapy and targeted gene delivery.

For his postdoctoral studies, Dr. Lisowski moved to Stanford University where he broadened his expertise in vector-based gene therapy approaches by mastering many aspects of adeno (Ad) and recombinant adeno-associated viral (rAAV) vector design, cloning, production and applications. His projects were focused on human embryonic stem cell specific rAAV vectors, in vivo gene therapy vector evolution via multispecies interbreeding, and retargeting of rAAV and novel rAAV vectors capable of non-random integration into the host genome. During his years at Stanford, he helped establish and served as a co-director of the rAAV core for Beta Cell Biology Consortium. As part of this effort, he established novel rAAV vector production protocols, which allow for production of high-titer, high-purity rAAV preparations.

Dr. Lisowski joined the Salk Institute in 2013 to direct and further develop the GT3 Core Facility. As part of GT3, he is improving and developing new protocols for LV, Ad, rAAV production, purification and titering. He is also working on establishing a large depository of AAV, LV and rabies-virus based reagents, which will be invaluable tools in projects related to President Obama's BRAIN Initiative, as well as in many other aspects of basic biological studies.


  • Medical University of Gdansk, Poland
  • BS in Biology, University of Bridgeport, Bridgeport, CT
  • PhD in Molecular Biology and Genetics, Weill Cornell Graduate School of Medical Sciences, New York, NY
  • Postdoctoral Fellow, Stanford University, Stanford, CA

Awards and Honors

  • Scientific Scholarship at the Academia Medica Gedanensis, Poland (1997)
  • University of Bridgeport Academic Excellence and Leadership Scholarship (1997-2001)
  • Dean's List and President's List (University of Bridgeport) (1997-1999)
  • National Collegiate Natural Science Award (1998, 1999)
  • Charles Reed Award, University of Bridgeport (1999, 2000)
  • Academic Achievement in Biology Award, University of Bridgeport (2000, 2001)
  • Travel Award. American Society of Gene & Cell Therapy (ASGCT) (2009, 2010, 2011)
  • Pediatric Research Fund Award. Stanford University (2009)
  • Walter V. and Idun Berry Pediatric Research Fellowship (2009-2013)

Selected Publications

  • Lisowski L, Elazar M, Chu K, Glenn J.S., Kay M.A., The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA. Nucleic Acid Research. 2013. PMID: 23396439
  • Valdmanis PN, Lisowski L, Kay MA., rAAV-mediated tumorigenesis: still unresolved after an AAV assault. Molecular Therapy, 2012, 20(11): 2014-7. PMID: 23131853. PMC Journal–In Process.
  • Lisowski L, Lau A, Wang Z, Zhang Y, Zhang F, Grompe M, Kay MA. Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. Molecular Therapy, 2012. 20(10):1912-23. PMID: 22990671. PMC Journal–In Process.
  • Wang Z, Lisowski L, Finegold MJ, Kay MA, Grompe M. AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Molecular Therapy,2012. 20(10):1902-11 PMID: 22990673. PMC Journal–In Process.
  • Wang Y, Zhang WY, Hu S, Lan F, Lee AS, Huber B, Lisowski L, Liang P, Huang M, de Almeida PE, Won JH, Sun N, Kay MA, Urnov FD, Wu JC. Human Embryonic and Induced Pluripotent Stem Cells Genome-Edited to Report on Their In Vivo Fate. Circulation Research, 2012. PMID:22967807
  • Sadelain M, Lisowski L, Chang A., Supplying therapeutic proteins from hematopoietic stem cell derived-erythroid and megakaryocytic lineage cells. Molecular Therapy, 2009; 17(12): 1994-9. PMCID: PMC2814379
  • Hayakawa J, Ueda T, Lisowski L, et al. Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the non-human primate. Human Gene Therapy, 2009 Feb 17. PMCID: PMC2828625
  • Sadelain M, Boulad F, Lisowski L, Moi P, Rivière I. Stem cell engineering for the treatment of severe hemoglobinopathies. Current Molecular Medicine. 2008; 8(7): 690-697.
  • Chang AH, Stephan MT, Lisowski L, Sadelain M. Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice. Molecular Therapy. 2008; 16(10): 1745-52. PMCID: PMC2658893
  • Lisowski L, Sadelain M. Current status of globin gene therapy for the treatment of ß-thalassemia. Br J Haematology, 2008; 114(3): 335-45. PMID: 18410569
  • Lisowski L, Sadelain M. Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in ß-thalassemic mice. Blood. 2007; 110(13): 4175-8. PMCID: PMC2234778
  • Samakoglu S, Lisowski L, Budak-Alpdogan T, Usachenko Y, Acuto S, Di Marzo R, Maggio A, Zhu P, Tisdale JF, Rivière I, Sadelain M. A genetic strategy to treat sickle cell anemia by co-regulating globin transgene expression and RNA interference. Nat Biotechnol. 2006; 24(1): 89-94. PMID: 16378095
  • Sadelain M, Lisowski L, Samakoglu S, Rivella S, May C, Rivière I. Progress toward the genetic treatment of the beta-thalassemias. Ann N Y Acad Sci. 2005; 1054: 78-91. PMID: 16339654
  • Sadelain M, Rivella S, Lisowski L, Samakoglu S, Rivière I. Globin gene transfer for treatment of the beta-thalassemias and sickle cell disease. Best Pract Res Clin Haematol. 2004; 17(3): 517-34. PMID:15498721
  • Rivella S, Lisowski L, Sadelain M. Globin gene transfer: a paradigm for transgene regulation and vector safety. Gene Therapy and Regulation 2003; 2(2): 149-175.

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