Finding cures for children's genetic diseases


Valdmanis PN, Gu S, Chu K, Jin L, Zhang F, Munding EM, Zhang Y, Huang Y, Kutay H, Ghoshal K, Lisowski L, Kay MA. RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice.Nature Medicine 2016 May;22(5):557-62
LisowskiL, Tay SS, Alexander IE. Adeno-associated virus serotypes for gene therapeutics. Curr Opin Pharmacol. 2015 Aug 17;24:59-67
Sebastiano V, Zhen HH, Haddad B, Bashkirova E, Melo SP, Wang P, Leung TL, Siprashvili Z, Tichy A, Li J, Ameen M, Hawkins J, Lee S, Li L, Schwertschkow A, Bauer G, LisowskiL, Kay MA, Kim SK, Lane AT, Wernig M, Oro AE. Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa. Science Translational Medicine. 2014, 6(264):264er8
Diecke S, Lisowski L, Kooreman, N. G., and Wu J.C., Second Generation Codon Optimized Minicircle (CoMiC) for Nonviral Reprogramming of Human Adult Fibroblasts. Methods in Molecular Biology, 2014, 1181: 1-13
Wang Y, Liang P, Lan F, Wu H, Lisowski L, Gu M, Hu S, Kay MA, Urnov FD, Shinnawi R, Gold JD, Gepstein L, Wu JC.Genome Editing of Isogenic Human Induced Pluripotent Stem Cells Recapitulates Long QT Phenotype for Drug Testing.Journalof theAmerican CollegeofCardiology. 2014 Aug 5;64(5):451-9
Melo SP, Lisowski L, Bashkirova E, Zhen HH, Chu K, Keene DR, Marinkovich MP, Kay MA, Oro AE., Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant. Molecular Therapy, 2014, 22(4): 725-33 (*co-first authors)
Lisowski L, Dane AP, Chu K, Zhang Y, Cunningham SC, Wilson EM, Nygaard S, Grompe M, Alexander IE, Kay MA.Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature, 2014, 506(7488): 382-6
Lisowski L, Elazar M, Chu K, Glenn JS, Kay MA.The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA. Nucleic Acid Research, 2013, 41(6): 3688-98
Wang N, Rajasekaran N, Hou T, Lisowski L, Mellins ED. Comparison of transduction efficiency among various lentiviruses containing GFP reporter in bone marrow hematopoietic stem cell transplantation. Exp Hematology, 2013, 41(11): 934-43
Valdmanis PN, Lisowski L, Kay MA, rAAV-mediated tumorigenesis: still unresolved after an AAV assault. Molecular Therapy, 2012, 20(11): 2014-7
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
Wang Z, Lisowski L, Finegold MJ, Nakai H, Kay MA, Grompe M.AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Molecular Therapy, 2012. 20(10): 1902-11
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, Robbins RC, Kay MA, Urnov FD, Wu JC. Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging. Circulation Research, 2012, 111(12):1494-503
Sadelain M, Chang A, Lisowski L., Supplying therapeutic proteins from hematopoietic stem cell derived-erythroid and megakaryocytic lineage cells. Molecular Therapy, 2009; 17(12): 1994-9
Hayakawa J, Ueda T, Lisowski L, Hsieh MM, Washington K, Phang O, Metzger M, Krouse A, Donahue RE, Sadelain M, Tisdale JF.Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the non-human primate. Human Gene Therapy, 2009; 20(6): 563-72
Sadelain M, Boulad F, Lisowski L, Moi P, Riviere 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
Lisowski L, Sadelain M. Current status of globin gene therapy for the treatment of β-thalassemia.British Journal of Haematology, 2008; 114(3): 335-45
Lisowski L, Sadelain M. Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in -thalassemic mice. Blood. 2007; 110(13): 4175-8
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 coregulating globin transgene expression and RNA interference. Nature Biotech. 2006; 24(1): 89-94
Sadelain M, Lisowski L, Samakoglu S, Rivella S, May C, Riviere I.Progress toward the genetic treatment of the beta-thalassemias.  Ann N Y Acad Sci. 2005; 1054: 78-91
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
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