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Josefina Piñón Hofbauer, PhD

Group Leader WG Piñón/Gruber

Research statement

Dr Piñón Hofbauer has 12 years experience in oncology research, including 5 years in EB cancer-related research. Her interests range from tumor biology and immunology to signaling pathways and mouse tumor models.
Her research currently focuses on the development of diagnostic assays and therapeutic strategies to combat epidermolysis bullosa-associated squamous cell carcinoma.

Personal interest

Loving mother, mad knitter, DIY buff, travel enthusiast, and closet karaoke aficionado.

Curriculum Vitae Josefina Piñón Hofbauer

Professional Career

Since 2014
Group leader
, EB House Austria

2013 - 2014
Senior Postdoc
, EB House Austria

2005 - 2013
Senior Postdoc
, Laboratory for Immunological and Molecular Cancer Research, Salzburg, Austria

2001 - 2005
, Biomedical Research Centre, Ninewells Hospital and Medical School, University of Dundee, Scotland

2000 - 2001
, Department of Microbiology, University of Pennsylvania, Philadelphia, PA, USA



in Molecular Biology (focus Microbiology/Virology), University of Pennsylvania School of Medicine, Philadelphia PA, USA

in Biology, Mount Holyoke College, South Hadley MA, USA


Awards and Fellowships

2016 Scientific award in bronze of the Paracelsus Medical University
2012 Scientific award in silver of the Paracelsus Medical University
2011 Sanofi Prize for Medical Research at the Paracelsus Medical University
2011 Scientific award in gold of the Paracelsus Medical University
2010 Wilhelm Türk Preis of the Austrian Socitey for Hematology and Oncology (ÖGHO)


Additional Qualifications

2017 EMBO Research Management course for Group Leaders
2015 Flow cytometry training for the Becton Dickinson LSR Fortessa
2010 Flow cytometry training for the Beckman Coulter Galios

Original Articles

  1. Hainzl S, Trattner L, Liemberger B, Bischof J, Kocher T, Ablinger M, Nyström A, Obermayer A, Klausegger A, Gruber C, Wally V, Bauer JW, Piñón Hofbauer J, Koller U. Splicing Modulation via Antisense Oligonucleotides in Recessive Dystrophic Epidermolysis Bullosa. International Journal of Molecular Sciences 2024.
  2. Brunner SM, Ramspacher A, Rieser C, Leitner J, Heil H, Ablinger M, Tevini J, Wimmer M, Koller A, Piñón Hofbauer J, Felder TK, Bauer JW, Kofler B, Lang R, Wally V. Topical Diacerein Decreases Skin and Splenic CD11c(+) Dendritic Cells in Psoriasis. International journal of molecular sciences 2023;24.
  3. Illmer J, Zauner R, Piñón Hofbauer J, Wimmer M, Gruner S, Ablinger M, Bischof J, Dorfer S, Hainzl S, Tober V, Bergson S, Sarig O, Samuelov L, Guttmann-Gruber C, Shalom-Feuerstein R, Sprecher E, Koller U, Laimer M, Bauer JW, Wally V. miR-200b-mediated reversion of a spectrum of epithelial to mesenchymal transition states in recessive dystrophic epidermolysis bullosa squamous cell carcinomas. The British journal of dermatology 2023.
  4. Zauner R, Wimmer M, Atzmueller S, Proell J, Niklas N, Ablinger M, Reisenberger M, Lettner T, Illmer J, Dorfer S, Koller U, Guttmann-Gruber C, Hofbauer JP, Bauer JW, Wally V. Biomarker Discovery in Rare Malignancies: Development of a miRNA Signature for RDEB-cSCC. 2023;15:3286.
  5. Welponer T, Weber DD, Trattner L, Tockner B, Aminzadeh-Gohari S, Leb-Reichl V, Kaufmann A, Zauner R, Wimmer M, Wally V, Felder TK, Strunk D, Koller U, Bauer JW, Kofler B, Guttmann-Gruber C, Piñon Hofbauer J. Metformin shows anti-neoplastic properties by inhibition of oxidative phosphorylation and glycolysis in epidermolysis bullosa-associated aggressive cutaneous squamous cell carcinoma. Journal of the European Academy of Dermatology and Venereology : JEADV 2023
  6. Liemberger B, Bischof J, Ablinger M, Hainzl S, Murauer EM, Lackner N, Ebner P, Kocher T, Nyström A, Wally V, Mayr E, Guttmann-Gruber C, Piñón Hofbauer J, Bauer JW,  Koller U. (2023). COL7A1 Editing via RNA Trans-Splicing in RDEB-Derived Skin Equivalents. Int J Mol Sci 24(5):4341.
  7. Zwicklhuber J, Kocher T, Liemberger B, Hainzl S, Bischof J, Strunk D, Raninger AM, Gratz I, Wally V, Guttmann-Gruber C, Piñón Hofbauer J, Bauer  JW, Koller  U (2023). A Novel Fluorescence-Based Screen of Gene Editing Molecules for Junctional Epidermolysis Bullosa. Int J Mol Sci 24(6):5197.
  8. Petković I, Bischof J, Kocher T, March OP, Liemberger B, Hainzl S, Strunk D, Raninger AM, Binder AM, Reichelt J, Guttman-Gruber C, Wally V, Piñón Hofbauer J, Bauer JW, Koller U. COL17A1 editing via homology-directed repair in junctional epidermolysis bullosa. Front Med 2022, 9:976604.
  9. Bischof J, March OP, Liemberger B, Haas SA, Hainzl S, Petković I, Leb-Reichl V, Illmer J, Korotchenko E, Klausegger A, Hoog A, Binder H-M, Garcia M, Duarte B, Strunk D, Larcher F, Reichelt J, Guttmann-Gruber C, Wally V, Hofbauer JP, Bauer JW, Cathomen T, Kocher T, Koller U. Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa. Molecular Therapy 2022.
  10. Mayr E, Ablinger M, Lettner T, Murauer EM, Guttmann-Gruber C, Piñón Hofbauer J, Hainzl S, Kaiser M, Klausegger A, Bauer JW, Koller U, Wally V. 5’ RNA Trans-Splicing Repair of COL7A1 Mutant Transcripts in Epidermolysis bullosa. Int J Mol Sci 2022, 23(3):1732.
  11. Zauner R, Wimmer M, Dorfer S, Ablinger M, Koller U, Piñón Hofbauer J, Guttmann-Gruber C, Bauer JW, Wally V. Transcriptome-Guided Drug Repurposing for Aggressive SCCs. International journal of molecular sciences 2022;23:1007
  12. Woess K, Sun Y, Morio H, Stierschneider A, Kaufmann A, Hainzl S, Trattner L, Kocher T, Tockner B, Leb-Reichl V, Steiner M, Brachtl G, South AP, Bauer JW, Reichelt J, Furihata T, Wally V, Koller U, Piñón Hofbauer J, Guttmann-Gruber C. Evaluating a Targeted Cancer Therapy Approach Mediated by RNA trans-Splicing In Vitro and in a Xenograft Model for Epidermolysis Bullosa-Associated Skin Cancer. International journal of molecular sciences 2022;23.
  13. Guttmann-Gruber C, Piñón Hofbauer J, Tockner B, Reichl V, Klausegger A, Hofbauer P, Wolkersdorfer M, Tham KC, Lim SS, Common JE, Diem A, Ude-Schoder K, Hitzl W, Lagler F, Reichelt J, Bauer JW, Lang R, Laimer M. Impact of low-dose calcipotriol ointment on wound healing, pruritus and pain in patients with dystrophic epidermolysis bullosa: A randomized, double-blind, placebo-controlled trial. Orphanet J Rare Dis 2021;16:473.
  14. Fuentes I, Guttmann-Gruber C, Tockner B, Diem A, Klausegger A, Cofré-Araneda G, Figuera O, Hidalgo Y, Morandé P, Palisson F, Rebolledo-Jaramillo B, Yubero MJ, Cho RJ, Rishel HI, Marinkovich MP, Teng JMC, Webster TG, Prisco M, Eraso LH, Piñon Hofbauer J, South AP. Cells from discarded dressings differentiate chronic from acute wounds in patients with Epidermolysis Bullosa. Scientific reports 2020;10:15064.
  15. Prodinger C, Diem A, Ude-Schoder K, Piñón-Hofbauer J, Kitzmueller S, Bauer JW, Laimer M. Profiling trial burden and patients’ attitudes to improve clinical research in epidermolysis bullosa. Orphanet Journal of Rare Diseases 2020;15:182
  16. Wimmer M, Zauner R, Ablinger M, Piñón-Hofbauer J, Guttmann-Gruber C, Reisenberger M, Lettner T, Niklas N, Proell J, Sajinovic M, De Souza P, Hainzl S, Kocher T, Murauer EM, Bauer JW, Strunk D, Reichelt J, Mellick AS, Wally V. A cancer stem cell-like phenotype is associated with miR-10b expression in aggressive squamous cell carcinomas. Cell Commun Signal 2020;18:61
  17. March OP, Lettner T, Klausegger A, Ablinger M, Kocher T, Hainzl S, Peking P, Lackner N, Rajan N, Hofbauer JP, Guttmann-Gruber C, Bygum A, Koller U, Reichelt J.  Gene editing-mediated disruption of epidermolytic ichthyosis-associated KRT10 alleles restores filament stability in keratinocytes. J Invest Dermatol. 2019 Aug;139(8):1699-1710.e6. doi: 10.1016/j.jid.2019.03.1146. Epub 2019 Apr 15.
  18. Sun Y*, Piñón Hofbauer J*, Harada M, Wöss K, Koller U, Morio H, Stierschneider A, Kitamura K, Hashimoto M, Chiba K, Akita H, Anzai N, Reichelt J, Bauer JW, Guttmann-Gruber C*, Furihata T*. Cancer-type organic anion transporting polypeptide 1B3 is a target for cancer suicide gene therapy using RNA trans-splicing technology. Cancer Lett. 2018 433:107-116. *equally contributing authors
  19. Fuentes I*, Guttmann-Gruber C*, Tay AS*, Piñón Hofbauer J, Denil S, Reichelt J, Palisson F, Common JE, South AP. Reduced microbial diversity is a feature of recessive dystrophic epidermolysis bullosa involved skin and wounds. J Invest Dermatol 2018 accepted manuscript. *equally contributing authors
  20. Liemberger B, Piñón Hofbauer J, Wally V, Arzt C, Hainzl S, Kocher T, Murauer EM, Bauer JW, Reichelt J, and Koller U. RNA Trans-Splicing Modulation via Antisense Molecule Interference. Int J Mol Sci 2018;19:762.
  21. Morio H, Sun Y, Harada M, Ide H, Shimozato O, Zhou X, Higashi K, Yuki R, Yamaguchi N, Piñón Hofbauer J, Guttmann-Gruber C, Anzai N, Akita H, Chiba K, and Furihata T. Cancer-Type OATP1B3 mRNA in Extracellular Vesicles as a Promising Candidate for a Serum-Based Colorectal Cancer Biomarker. Biol Pharm Bull 2018;41:445-449.
  22. Sun Y, Woess K, Kienzl M, Leb-Reichl VM, Feinle A, Wimmer M, Zauner R, Wally V, Luetz-Meindl U, Mellerio JE, Fuentes I, South AP, Bauer JW, Reichelt J, Furihata T, Guttmann-Gruber C, and Piñón Hofbauer J. Extracellular Vesicles as Biomarkers for the Detection of a Tumor Marker Gene in Epidermolysis Bullosa-Associated Squamous Cell Carcinoma. J Invest Dermatol 2017;12
  23. Kocher T, Peking P, Klausegger A, Murauer EM, Piñón Hofbauer J, Wally V, Lettner T, Hainzl S, Ablinger M, Bauer JW, Reichelt J, and Koller U. Cut and Paste: Efficient Homology-Directed Repair of a Dominant Negative KRT14 Mutation via CRISPR/Cas9 Nickases. Mol Ther 2017;25:2585-2598.
  24. Atanasova VS, Jiang Q, Prisco M, Gruber C, Piñón Hofbauer J, Chen M, Has C, Bruckner-Tuderman L, McGrath JA, Uitto J, and South AP. Amlexanox Enhances Premature Termination Codon Read-Through in COL7A1 and Expression of Full Length Type VII Collagen: Potential Therapy for Recessive Dystrophic Epidermolysis Bullosa. J Invest Dermatol 2017;137:1842-1849.
  25. Zaborsky N, Gassner FJ, Asslaber D, Reinthaler P, Denk U, Flenady S, Piñón Hofbauer J, Danner B, Rebhandl S, Harrer A, Geisberger R, Greil R, and Egle A. CD1d expression on chronic lymphocytic leukemia B cells affects disease progression and induces T cell skewing in CD8 positive and CD4CD8 double negative T cells. Oncotarget 2016;7:49459-49469.
  26. Kocher T, Asslaber D, Zaborsky N, Flenady S, Denk U, Reinthaler P, Ablinger M, Geisberger R, Bauer JW, Seiffert M, Hartmann TN, Greil R, Egle A, and Piñón Hofbauer J. CD4+ T cells, but not non-classical monocytes, are dispensable for the development of chronic lymphocytic leukemia in the TCL1-tg murine model. Leukemia 2016;30:1409-1413.
  27. Hofbauer SW, Krenn PW, Piñón Hofbauer J, Pucher S, Asslaber D, Egle A, Hartmann TN, Greil R. The AKT1 isoform plays a dominant role in the survival and chemoresistance of chronic lymphocytic leukaemia cells. Br J Haematol 2016;172(5):815-9.
  28. Zaborsky N, Holler C, Geisberger R, Asslaber D, Gassner FJ, Egger V, Piñón Hofbauer J, Kocher T, Hartmann TN, Greil R, and Egle A. B-cell receptor usage correlates with the sensitivity to CD40 stimulation and the occurrence of CD4+ T-cell clonality in chronic lymphocytic leukemia. Haematologica 2015;100:e307-e310.
  29. Grössinger E,Weiss L, Zierler S, Rebhandl S, Krenn PW, Hinterseer E, Schmölzer J, Asslaber D, Hainzl S, Neureiter D, Egle A, Piñón Hofbauer J, Hartmann TN, Greil R, and Kerschbaum H. Targeting proliferation of chronic lymphocytic leukemia (CLL) cells through KCa3.1 blockade. Leukemia 2014;28:954-958.
  30. Valente LJ, Gray DHD, Michalak EM, Piñón Hofbauer J, Egle A, Scott CL, Janic A, and Strasser A. p53 efficiently suppresses tumor development in the complete absence of its cell-cycle inhibitory and proapoptotic effectors p21, Puma, and Noxa. Cell Rep 2013;3:1339-1345.
  31. Merkel O, Wacht N, Sifft E, Melchardt T, Hamacher F, Kocher T, Denk U, Piñón Hofbauer J, Egle A, Scheideler M, Schlederer M, Steurer M, Kenner L, and Greil R. Actinomycin D induces p53-independent cell death and prolongs survival in high-risk chronic lymphocytic leukemia. Leukemia 2012;26:2508-2516.
  32. Piñón Hofbauer J, Heyder C, Denk U, Kocher T, Holler C, Trapin D, Asslaber D, Tinhofer I, Greil R, and Egle A. Development of CLL in the TCL1 transgenic mouse model is associated with severe skewing of the T-cell compartment homologous to human CLL. Leukemia 2011;25:1452-1458.
  33. Gassner FJ, Weiss L, Geisberger R, Piñón Hofbauer J, Egle A, Hartmann TN, Greil R, and Tinhofer I. Fludarabine modulates composition and function of the T cell pool in patients with chronic lymphocytic leukaemia. Cancer Immunol Immunother 2011;60:75-85.
  34. Brachtl G, Sahakyan K, Denk U, Girbl T, Alinger B, Hofbauer SW, Neureiter D, Piñón Hofbauer J, Egle A, Greil R, and Hartmann TN. Differential bone marrow homing capacity of VLA-4 and CD38 high expressing chronic lymphocytic leukemia cells. PloS one 2011;6:e23758.
  35. Hofbauer SW, Piñón JD, Brachtl G, Haginger L, Wang W, Jöhrer K, Tinhofer I, Hartmann TN, and Greil R. Modifying akt signaling in B-cell chronic lymphocytic leukemia cells. Cancer Res 2010;70:7336-7344.
  36. Merkel O, Asslaber D, Piñón JD, Egle A, and Greil R. Interdependent regulation of p53 and miR-34a in chronic lymphocytic leukemia. Cell cycle 2010;9:2764-2768.
  37. Asslaber D, Piñón JD, Seyfried I, Desch P, Stöcher M, Tinhofer I, Egle A, Merkel O, and Greil R. microRNA-34a expression correlates with MDM2 SNP309 polymorphism and treatment-free survival in chronic lymphocytic leukemia. Blood 2010;115:4191-4197.
  38. Holler C, Piñón JD, Denk U, Heyder C, Hofbauer S, Greil R, and Egle A. PKCbeta is essential for the development of chronic lymphocytic leukemia in the TCL1 transgenic mouse model: validation of PKCbeta as a therapeutic target in chronic lymphocytic leukemia. Blood 2009;113:2791-2794.
  39. Piñón JD, Klasse PJ, Jassal SR, Welson S, Weber J, Brighty DW, and Sattentau QJ. Human T-cell leukemia virus type 1 envelope glycoprotein gp46 interacts with cell surface heparan sulfate proteoglycans. J Virol 2003;77:9922-9930.
  40. Piñón JD, Kelly SM, Price NC, Flanagan JU, and Brighty DW. An antiviral peptide targets a coiled-coil domain of the human T-cell leukemia virus envelope glycoprotein. J Virol 2003;77:3281-3290.
  41. Tsai JC, Groot L de, Piñón JD, Iacono KT, Phillips JJ, Seo Sh, Lavi E, and Weiss SR. Amino acid substitutions within the heptad repeat domain 1 of murine coronavirus spike protein restrict viral antigen spread in the central nervous system. Virology 2003;312:369-380.
  42. De Groot L, Piñón JD, Phillips J, Lavi E, and Weiss SR. Pathogenesis of fusion deficient recombinant mouse hepatitis viruses. Adv Exp Med Biol 2001;494:159-162.
  43. Piñón JD, Teng H, and Weiss SR. Further requirements for cleavage by the murine coronavirus 3C-like proteinase: identification of a cleavage site within ORF1b. Virology 1999;263:471-484.
  44. Teng H, Piñón JD, and Weiss SR. Expression of murine coronavirus recombinant papain-like proteinase: efficient cleavage is dependent on the lengths of both the substrate and the proteinase polypeptides. J Virol 1999;73:2658-2666.
  45. Bi W, Piñón JD, Hughes S, Bonilla PJ, Holmes KV, Weiss SR, and Leibowitz JL. Localization of mouse hepatitis virus open reading frame 1A derived proteins. J Neurovirol 1998;4:594-605.
  46. Piñón JD, Mayreddy RR, Turner JD, Khan FS, Bonilla PJ, and Weiss SR. Efficient autoproteolytic processing of the MHV-A59 3C-like proteinase from the flanking hydrophobic domains requires membranes. Virology 1997;230:309-322.
  47. Bonilla PJ, Hughes SA, Piñón JD, and Weiss SR. Characterization of the leader papain-like proteinase of MHV-A59: identification of a new in vitro cleavage site. Virology 1995;209:489-497.

Book Chapters

  1. Piñón Hofbauer J, Wally V, Guttmann-Gruber C, Gratz IK, Koller U. Therapy Development for Epidermolysis Bullosa [Online First].  Rare Diseases: Intechopen; 2021.
  2. Wagner RN, Piñón Hofbauer J, Wally V, Kofler B, Schmuth M, De Rosa L, De Luca M, Bauer JW. Epigenetic and metabolic regulation of epidermal homeostasis. Exp Dermatol 2021;30:1009-22.


  1. Piñón Hofbauer J, Guttmann-Gruber C, Wally V, Sharma A, Gratz IK, Koller U. Challenges and progress related to gene editing in rare skin diseases. Adv Drug Deliv Rev 2024;208:115294.
  2. Guttmann-Gruber C, Bauer JW, Piñón Hofbauer J. Hereditary bullous diseases: current and innovative models to study the skin blistering disease epidermolysis bullosa. Drug Discovery Today: Disease Models 2020.
  3. Brachtl G, Piñón Hofbauer J, Greil R, Hartmann TN. The pathogenic relevance of the prognostic markers CD38 and CD49d in chronic lymphocytic leukemia. Ann Hematol 2014;93(3):361-74.
  4. Piñón JD, Labi V, Egle A, and Villunger A. Bim and Bmf in tissue homeostasis and malignant disease. Oncogene 2008;27 Suppl 1:S41-S52.

Scientific Commentaries

  1. Gruber C, Piñón Hofbauer J, and Bauer JW. Can we be SMaRT-er in our approach to cancer therapy? EBioMedicine 2015;2. Original DateCompleted: 20150820:621-622.
  2. Egle A, Asslaber D, Villunger A, and Piñón Hofbauer J. Bid-ding for mercy: twisted killer in action. Cell Death Differ 2013;20:847-849.


  1. Guttmann-Gruber C, Tockner B, Scharler C, Hüttner C, Common JE, Tay ASL, Denil SLIJ, Klausegger A, Trost A, Breitenbach J, Schnitzhofer P, Hofbauer JP, Wolkersdorfer M, Diem A, Laimer M, Strunk D, Bauer JW, Reichelt J, Lang R, Piñón Hofbauer J. VD3-Analog Calcipotriol verbessert Wundheilungsprozess. Spectrum Dermatologie 2019;02/2019.
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