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Nicole Paulk, PhD

TitleAssistant Professor
InstitutionUniversity of California San Francisco
DepartmentBiochemistry and Biophysics
Address1700 4th St
San Francisco CA 94158
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    Collapse Biography 
    Collapse Education and Training
    Stanford UniversityPostdoc08/2016Human Gene Therapy
    Oregon Health & Science UniversityPhD06/2012Viral Gene Therapy and Liver Regeneration
    Central Washington UniversityBS06/2006Medical Microbiology

    Collapse Overview 
    Collapse Overview
    For patients seeking treatment for genetic diseases, the standard of care can often be invasive (e.g. liver transplantation) or temporary (e.g. enzyme replacement therapy). Gene and cell therapies are attractive alternatives as they can be non-invasive with long-term therapeutic benefits. However, the success of this new generation of translational therapies is critically dependent on efficiency, efficacy, safety and cost. In the Paulk lab we engineer new technologies to address these needs. We develop viral genome engineering platforms, design combinatorial gene therapies to improve targeting and reduce costs, study sexually dimorphic responses to viral transduction and tissue regeneration, and create new human liver mouse models to validate responses to gene and cell therapies in vivo.


    Collapse Research 
    Collapse Research Activities and Funding
    Human microtissues for in vitro detection and functional measurement of adverse consequences caused by genome editing
    NIH/OD U01-HL145795Sep 25, 2018 - Jun 30, 2023
    Role: Co-Investigator
    Development of Experimental Viral Gene Therapies for Glioblastoma.
    GLIOMA PRECISION MEDICINE PROGRAM May 1, 2018 - Jun 1, 2019
    Role: Co-PI
    Sexually dimorphic viral-host interactions in pediatric liver diseases.
    STANFORD UNIVERSITY INSTITUTE FOR IMMUNITY, TRANSPLANTATION & INFECTION Nov 1, 2016 - Jun 22, 2017
    Role: Principal Investigator
    Overcoming sexually dimorphic barriers to viral gene therapy for treating genetic liver diseases
    NIH/NIDDK K01DK107607Aug 4, 2016 - May 31, 2021
    Role: Principal Investigator
    Characterizing whether clinical and research-grade rAAV vectors differ in post-translational modifications.
    STANFORD UNIVERSITY MASS SPECTROMETRY CORE FACILITY Sep 1, 2015 - Aug 31, 2016
    Role: Co-PI
    Novel strategies to expand the adeno-associated virus platform.
    NIH/NHLBI F32HL119059Jun 19, 2014 - Jun 18, 2017
    Role: Principal Investigator
    Determining the Progenitor-Like Capacity of Liver Cancer Cells in vivo.
    NIH/NCI F31CA130116Sep 1, 2007 - Jun 30, 2012
    Role: Principal Investigator

    Collapse Bibliographic 
    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help.
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    1. Paulk N, Pekrun K, Charville G, Nguyen K, Wosczyna M, Xu J, Zhang Y, Yoo B, Moure J, Lee G, Shrager J, Rando T, and Kay M.Bioengineered viral platform for intramuscular passive vaccine delivery to human skeletal muscle. Molecular Therapy Methods & Clinical Development. 2018; 10:144-155.
      View Publication
    2. Paulk NK, Pekrun K, Charville GW, Maguire-Nguyen K, Wosczyna MN, Xu J, Zhang Y, Lisowski L, Yoo B, Vilches-Moure JG, Lee GK, Shrager JB, Rando TA, Kay MA. Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle. Mol Ther Methods Clin Dev. 2018 Sep 21; 10:144-155. PMID: 30101152.
      View in: PubMed
    3. Winters IP, Chiou SH, Paulk NK, McFarland CD, Lalgudi PV, Ma RK, Lisowski L, Connolly AJ, Petrov DA, Kay MA, Winslow MM. Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity. Nat Commun. 2017 Dec 12; 8(1):2053. PMID: 29233960.
      View in: PubMed
    4. Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid a-glucosidase. Sci Transl Med. 2017 Nov 29; 9(418). PMID: 29187643.
      View in: PubMed
    5. Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA. Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. Mol Ther. 2018 Jan 03; 26(1):289-303. PMID: 29055620.
      View in: PubMed
    6. Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature. 2015 Jan 15; 517(7534):360-4. PMID: 25363772; PMCID: PMC4297598.
      View in: PubMed, PubMed Central
    7. Paulk NK, Wursthorn K, Haft A, Pelz C, Clarke G, Newell AH, Olson SB, Harding CO, Finegold MJ, Bateman RL, Witte JF, McClard R, Grompe M. In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice. Mol Ther. 2012 Oct; 20(10):1981-7. PMID: 22871666; PMCID: PMC3464641.
      View in: PubMed, PubMed Central
    8. Paulk NK, Loza LM, Finegold MJ, Grompe M. AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo. Hum Gene Ther. 2012 Jun; 23(6):658-65. PMID: 22486314; PMCID: PMC3392621.
      View in: PubMed, PubMed Central
    9. Taylor AM, Preston AJ, Paulk NK, Sutherland H, Keenan CM, Wilson PJ, Wlodarski B, Grompe M, Ranganath LR, Gallagher JA, Jarvis JC. Ochronosis in a murine model of alkaptonuria is synonymous to that in the human condition. Osteoarthritis Cartilage. 2012 Aug; 20(8):880-6. PMID: 22542924; PMCID: PMC3406176.
      View in: PubMed, PubMed Central
    10. Paulk NK.. Novel strategies to improve viral gene targeting and therapeutic liver repopulation in vivo. 2012.
      View Publication
    11. Paulk NK, Wursthorn K, Wang Z, Finegold MJ, Kay MA, Grompe M. Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology. 2010 Apr; 51(4):1200-8. PMID: 20162619; PMCID: PMC3136243.
      View in: PubMed, PubMed Central
    12. Duncan AW, Hickey RD, Paulk NK, Culberson AJ, Olson SB, Finegold MJ, Grompe M. Ploidy reductions in murine fusion-derived hepatocytes. PLoS Genet. 2009 Feb; 5(2):e1000385. PMID: 19229314; PMCID: PMC2636893.
      View in: PubMed, PubMed Central
    13. Key J, Paulk N, and Johansen A.Effects of DMSO oxidation products on iron speciation in photochemical simulation experiments. Environ Sci Technol. 2008; 1(42):133-139.
      View Publication
    14. Azuma H, Paulk N, Ranade A, Dorrell C, Al-Dhalimy M, Ellis E, Strom S, Kay MA, Finegold M, Grompe M. Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice. Nat Biotechnol. 2007 Aug; 25(8):903-10. PMID: 17664939; PMCID: PMC3404624.
      View in: PubMed, PubMed Central
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