Nicole Paulk, PhD

Title(s)Affiliate, HDF Comprehensive Cancer Ctr
SchoolSchool of Medicine
Address1700 4th Street, #404
San Francisco CA 94158
ORCID ORCID Icon0000-0002-5557-6280 Additional info
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    Collapse Biography 
    Collapse Education and Training
    Stanford UniversityPostdoc08/2016Human Gene Therapy
    Oregon Health & Science UniversityPhD06/2012Viral Gene Therapy
    Central Washington UniversityBS06/2006Medical Microbiology

    Collapse Overview 
    Collapse Overview
    For patients seeking treatment for rare genetic diseases and cancer, the standard of care can often be invasive (e.g. organ transplantation or surgical debulking) and temporary (e.g. enzyme replacement therapy or chemotherapy). AAV gene therapies are attractive alternatives as they are non-invasive with long-term therapeutic benefits. However, the success of this new generation of transformative therapies is critically dependent on successful delivery, strong efficacy, improved safety and reduced cost. In the Paulk lab we engineer disruptive technologies to directly address each of these to make AAV cheaper, easier and faster to get these life-saving drugs to the patients who need them.

    Dr. Paulk is an Associate Member of UCSF Neurologic Oncology, Member of the UCSF Helen Diller Family Comprehensive Cancer Center, Member of the UCSF Glioblastoma Precision Medicine Program, and Member of the UCSF Liver Center.
    Collapse In The News

    Collapse Research 
    Collapse Research Activities and Funding
    A Novel Gene Therapy Platform for Glioblastoma
    UCSF CATALYST PROGRAM Jun 1, 2020 - Jun 1, 2021
    Role: Principal Investigator
    Characterizing the proteomic landscape of rAAV vectors for human liver gene therapies.
    AMERICAN SOCIETY OF GENE & CELL THERAPY Dec 12, 2018 - Dec 11, 2019
    Role: Principal Investigator
    Human microtissues for in vitro detection and functional measurement of adverse consequences caused by genome editing.
    NIH/NIBIB U01-EB029374Sep 25, 2018 - Jun 30, 2023
    Role: Co-Investigator
    Development of Experimental Viral Gene Therapies for Glioblastoma.
    Role: Co-PI
    Sexually dimorphic viral-host interactions in pediatric liver diseases.
    Role: Principal Investigator
    Overcoming sexually dimorphic barriers to viral gene therapy for treating genetic liver diseases
    NIH K01DK107607Aug 4, 2016 - May 31, 2021
    Role: Principal Investigator
    Characterizing whether clinical and research-grade rAAV vectors differ in post-translational modifications.
    Role: Co-PI
    Novel strategies to expand the adeno-associated virus platform.
    NIH F32HL119059Jun 19, 2014 - Jun 18, 2017
    Role: Principal Investigator
    Determining the Progenitor-Like Capacity of Liver Cancer Cells in vivo.
    NIH F31CA130116Sep 1, 2007 - Jun 30, 2012
    Role: Principal Investigator

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    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. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Comparison of highly pure rAAV9 vector stocks produced in suspension by PEI transfection or HSV infection reveals striking quantitative and qualitative differences. Mol Ther Methods Clin Dev. 2022 Mar 10; 24:154-170. Trivedi PD, Yu C, Chaudhuri P, Johnson EJ, Caton T, Adamson L, Byrne BJ, Paulk NK, Clément N. PMID: 35071688; PMCID: PMC8760416.
      View in: PubMed   Mentions: 6  
    2. Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics". Front Immunol. 2021; 12:822389. Martinez-Navio JM, Paulk NK, Gao G. PMID: 34992614; PMCID: PMC8724200.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansAnimalsCells
    3. Editorial: “AAV Gene Therapy: Immunology and Immunotherapeutics”. Frontiers in Immunology. 2021 Dec 21; 12:822389. Martinez-Navio MJ, Paulk PN, Gao GG. .
      View in: Publisher Site   Mentions:
    4. VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector Impurities. Front Immunol. 2021; 12:657795. Rumachik NG, Malaker SA, Paulk NK. PMID: 33868302; PMCID: PMC8047074.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCells
    5. Methods Matter -- Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors. Molecular Therapy - Methods & Clinical Development. 2020 Sep 1; 18:98-118. Rumachik RN, Malaker MS, Poweleit PN, Maynard ML, Adams AC, Leib LR, Cirolia CG, Thomas TD, Stamnes SS, Holt HK, Sinn SP, May MA, Paulk PN. .
      View in: Publisher Site   Mentions:
    6. Gene Therapy: It Is Time to Talk about High-Dose AAV. Genetic Engineering & Biotechnology News. 2020 Sep 1; 40(9):14-16. Paulk PN. .
      View in: Publisher Site   Mentions:
    7. Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors. Mol Ther Methods Clin Dev. 2020 Sep 11; 18:98-118. Rumachik NG, Malaker SA, Poweleit N, Maynard LH, Adams CM, Leib RD, Cirolia G, Thomas D, Stamnes S, Holt K, Sinn P, May AP, Paulk NK. PMID: 32995354; PMCID: PMC7488757.
      View in: PubMed   Mentions: 49  
    8. Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. Hum Gene Ther Methods. 2019 12; 30(6):195-205. Maynard LH, Smith O, Tilmans NP, Tham E, Hosseinzadeh S, Tan W, Leenay R, May AP, Paulk NK. PMID: 31855083; PMCID: PMC6919253.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansAnimalsCells
    9. An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging. Nat Chem Biol. 2019 05; 15(5):433-436. Oh Y, Park Y, Cho JH, Wu H, Paulk NK, Liu LX, Kim N, Kay MA, Wu JC, Lin MZ. PMID: 30936501; PMCID: PMC6563924.
      View in: PubMed   Mentions: 21     Fields:    Translation:Animals
    10. Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle. Mol Ther Methods Clin Dev. 2018 Sep 21; 10:144-155. 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. PMID: 30101152; PMCID: PMC6077147.
      View in: PubMed   Mentions: 12  
    11. Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity. Nat Commun. 2017 12 12; 8(1):2053. Winters IP, Chiou SH, Paulk NK, McFarland CD, Lalgudi PV, Ma RK, Lisowski L, Connolly AJ, Petrov DA, Kay MA, Winslow MM. PMID: 29233960; PMCID: PMC5727199.
      View in: PubMed   Mentions: 47     Fields:    Translation:AnimalsCells
    12. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. Sci Transl Med. 2017 Nov 29; 9(418). 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. PMID: 29187643; PMCID: PMC5826611.
      View in: PubMed   Mentions: 75     Fields:    Translation:AnimalsCells
    13. Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. Mol Ther. 2018 01 03; 26(1):289-303. 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. PMID: 29055620; PMCID: PMC5763027.
      View in: PubMed   Mentions: 87     Fields:    Translation:HumansAnimalsCells
    14. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature. 2015 Jan 15; 517(7534):360-4. Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA. PMID: 25363772; PMCID: PMC4297598.
      View in: PubMed   Mentions: 142     Fields:    Translation:HumansAnimalsCells
    15. In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice. Mol Ther. 2012 Oct; 20(10):1981-7. 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. PMID: 22871666; PMCID: PMC3464641.
      View in: PubMed   Mentions: 7     Fields:    Translation:AnimalsCells
    16. 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. Paulk NK, Loza LM, Finegold MJ, Grompe M. PMID: 22486314; PMCID: PMC3392621.
      View in: PubMed   Mentions: 16     Fields:    Translation:AnimalsCells
    17. Ochronosis in a murine model of alkaptonuria is synonymous to that in the human condition. Osteoarthritis Cartilage. 2012 Aug; 20(8):880-6. Taylor AM, Preston AJ, Paulk NK, Sutherland H, Keenan CM, Wilson PJ, Wlodarski B, Grompe M, Ranganath LR, Gallagher JA, Jarvis JC. PMID: 22542924; PMCID: PMC3406176.
      View in: PubMed   Mentions: 15     Fields:    Translation:AnimalsCells
    18. Novel strategies to improve viral gene targeting and therapeutic liver repopulation in vivo. 2012. Paulk NK. View Publication.
    19. Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology. 2010 Apr; 51(4):1200-8. Paulk NK, Wursthorn K, Wang Z, Finegold MJ, Kay MA, Grompe M. PMID: 20162619; PMCID: PMC3136243.
      View in: PubMed   Mentions: 59     Fields:    Translation:AnimalsCells
    20. Ploidy reductions in murine fusion-derived hepatocytes. PLoS Genet. 2009 Feb; 5(2):e1000385. Duncan AW, Hickey RD, Paulk NK, Culberson AJ, Olson SB, Finegold MJ, Grompe M. PMID: 19229314; PMCID: PMC2636893.
      View in: PubMed   Mentions: 52     Fields:    Translation:AnimalsCells
    21. Effects of DMSO oxidation products on iron speciation in photochemical simulation experiments. Environ Sci Technol. 2008; 1(42):133-139. Key J, Paulk N, and Johansen A. View Publication.
    22. Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice. Nat Biotechnol. 2007 Aug; 25(8):903-10. Azuma H, Paulk N, Ranade A, Dorrell C, Al-Dhalimy M, Ellis E, Strom S, Kay MA, Finegold M, Grompe M. PMID: 17664939; PMCID: PMC3404624.
      View in: PubMed   Mentions: 370     Fields:    Translation:HumansAnimalsCells
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