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Sushrusha Nayak

Affiliated to research

Department and organisational affiliation:

About me

I am affiliated with the Department of medicine in Karolinska institute. My interest is in translational medicine. The study and use of regulatory T cells (Treg) for immune regulation in gene therapy and protein replacement therapy. To reduce the incidence of drug induced immune responses in therapies for lysosomal storage diseases like Pompe, enzyme deficiencies like hemophilia. 

My current project involves the development of mehodology to move single cell sequencing to the clinic, including the prediction of immune responses in protein relacement therapies. 

Research description


Doerfler PA, Nayak S, Corti M, Morel L, Herzog RW, Byrne BJ. Targeted approaches to induce immune tolerance for Pompe disease therapy. Mol Ther Methods Clin Dev. 2016 Jan 27;3:15053.

Doerfler PA, Todd AG, Clement N, Falk D, Nayak S, Herzog R, Byrne BJ. Co-Packaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Hum Gene Ther. 2015 Nov 25. [Epub ahead of print]

Biswas M, Sarkar D, Kumar SR, Nayak S, Rogers GL, Markusic DM, Liao G, Terhorst C, Herzog RW. Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell-dependent induction of CD4+CD25+FoxP3+ Treg. Blood. 2015 Apr 1. pii: blood-2014-09-599266. 

Doerfler PA, Nayak S, Herzog RW, Morel L, Byrne BJ. BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease. Clin Immunol. 2015 Apr 1. pii: S1521-6616(15)00125-4. 

Nayak S, Doerfler PA, Porvasnik SL, Cloutier DD, Khanna R, Valenzano KJ, Herzog RW, Byrne BJ: Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent. PloS one 2014, 9:e98336. doi:  10.1371/journal.pone.0098336

Corti M, Elder M, Falk D, Lawson L, Smith B, Nayak S, Conlon T, Clement N, Erger K, Lavassani E, et al: B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study. Mol Ther Methods Clin Dev. 2014; 1: 14033. doi:  10.1038/mtm.2014.33

Elmallah MK, Falk DJ, Nayak S, Federico RA, Sandhu MS, Poirier A, Byrne BJ, Fuller DD. Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice. Mol Ther Methods Clin Dev. 2014;1. pii: 14033.

Manganese superoxide dismutase promotes anoikis resistance and tumor metastasis. Kamarajugadda S, Cai Q, Chen H, Nayak S, Zhu J, He M, Jin Y, Zhang Y, Ai L, Martin SS, Tan M, Lu J. Cell Death Dis. 2013 Feb 21;4:e504. 

Markusic DM1, Hoffman BE, Perrin GQ, Nayak S, Wang X, LoDuca PA, High KA, Herzog RW. Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies. EMBO Mol Med. 2013 Nov;5(11):1698-709. 

Nayak S*, Elder M*, Lawson L, Collins S, Harfe K, Byrne BJ. Immune Modulation in CRIM- Patients with Early Onset Pompe Disease. J Pediatr. 2013 Sep;163(3):847-54.e1.

Nayak S*, Sivakumar R*, Cao O, BJ Byrne, RW Herzog. Multiple CD4+ T Cell Epitopes Contribute to the Immune Response to Acid a-Glucosidase in Pompe Mice. Mol Genet Metab. 2012 Jun;106(2):189-95. Epub 2012 Mar 23.

Kamarajugadda S, Stemborroski L, Simpson NE, Nayak S, Tan M, Lu J. Glucose oxidation modulates anoikis and tumor metastasis. Mol Cell Biol. 2012 May;32(10):1893-907.

Nayak S, Sarkar D, Moghimi B, Perrin GQ, Zhou S, Byrne BJ, Herzog RW. Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B. 2011. Front Microbiol.2011;2:244.

Moghimi B, Sack BK, Nayak S, Markusic DM, Mah CS, Herzog RW. Tolerance Induction to Factor VIII by Transient Co-administration with Rapamycin. J Thromb Haemost. 2011 Aug;9(8):1524-33. 

Byrne BJ, Falk DJ, Pacak CA, Nayak S, Herzog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, Smith BK, Fuller DD, Lawson LA, Mah CS. Pompe Disease Gene Therapy. Hum Mol Genet. 2011 Apr 15;20(R1):R61-8.

Liao G, Nayak S, Regueiro JR, Berger SB, Detre C, Romero X, de Waal Malefyt R, Chatila TA, Herzog RW, Terhorst C. GITR engagement preferentially enhances proliferation of functionally competent CD4+CD25+FoxP3+ regulatory T cells. Int Immunol. 2010 Apr;22(4):259-70. Epub 2010 Feb 5

Nayak S and Herzog RW. Progress and Prospects: Immune Responses to Viral Vectors. Invited Review. Gene Ther. 2010 Mar;17(3):295-304. Epub 2009 Nov 12

Nayak S, Cao O, Hoffman BE, Cooper M, Zhou S, Atkinson MA, Herzog RW. Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cellsJ Thromb Haemost. 2009 Sep;7(9):1523-32. Epub 2009 Jul 6

Nayak S*, Martino AT*, Hoffman BE, Cooper M, Liao G, et al. (2009) Tolerance Induction to Cytoplasmic b-Galactosidase by Hepatic AAV Gene Transfer — Implications for Antigen Presentation and Immunotoxicity. PLoS ONE 4(8): e6376

Cooper M, Nayak S, Hoffman BE, Terhost C, Cao O, Herzog RW. Improved Induction of Immune Tolerance to Factor IX by Hepatic AAV-8 Gene Transfer. Human Gene Therapy. July 2009, 20(7): 767-776.

Cao O, Hoffman BE, Moghimi B, Nayak S, Cooper M, Zhou S, Hildegund CJ Ertl, High KA and Herzog RW. Impact of the Underlying Mutation and the Route of Vector Administration on Immune Responses to Factor IX in Gene Therapy for Hemophilia B. Mol Ther. 2009 Oct;17(10):1733-42.

Cao O, Dobrzynski E, Wang L, Nayak S, Mingle B, Terhost C, Herzog RW. Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood 110: 1132-1140. (2007)


Doctorate in Gene Therapy and Immunology (PhD): University of Florida, College of Medicine, Gainesville, USA, 2010

Master of Science in Management (MSM): Hough School of Business, University of Florida, Gainesville, USA, 2009

Master of Science in Biotechnology (MSc): Bangalore University, Bangalore, India, 2005

Bachelors in Microbiology, Chemistry and Zoology (BSc): Bangalore University, Bangalore, India 2003

Academic honours, awards and prizes

Selected Research Awards:

1999 Gold-All India Jawaharlal Nehru National Science Exhibition, Lucknow, India 

2010 Graduate Student Council Research Award, University of Florida, FL, USA

2015 Kronprinsessan Lovisas Foundation for Child Health, Sweden 2015 Swedish Society of Medicine, Sweden

Fellowship Awards:

2008 NIH T32 NRSA Training Grant Pre-doctoral Fellowship; Regenerative Medicine, University of Florida, FL, USA

2014, 2015 Wenner-Gren Postdoctoral Fellowship, Stockholm, Sweden 


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