Nicole Paulk, PhD
Affiliate
HDF Comprehensive Cancer Ctr
School of Medicine
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.
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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.
Education & Training
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- Postdoc Human Gene Therapy Stanford University 08/2016
- PhD Viral Gene Therapy Oregon Health & Science University 06/2012
- BS Medical Microbiology Central Washington University 06/2006
Interests
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- Gene Therapy
- Genomics
- Bioengineering
- Rare Disease
- Immunotherapy
- AAV
- Proteomics
- Epigenomics
- Sexual Dimorphism
- Cancer
- Technology Development
Websites
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- @@Nicole_Paulk on Twitter (twitter.com)
- Paulk Lab GitHub Code (github.com)
- Paulk Lab Addgene Plasmids (addgene.org)
- Paulk Lab Protocols.io (protocols.io)
- Nicole Paulk ResearchGate (researchgate.net)
- Nicole Paulk ORCID (orcid.org)
- Nicole Paulk LinkedIn (linkedin.com)
Grants and Projects
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- Human microtissues for in vitro detection and functional measurement of adverse consequences caused by genome editing., NIH/NIBIB, 2018-2023
- A Novel Gene Therapy Platform for Glioblastoma, UCSF CATALYST PROGRAM, 2020-2021
- Overcoming sexually dimorphic barriers to viral gene therapy for treating genetic liver diseases, NIH, 2016-2021
- Characterizing the proteomic landscape of rAAV vectors for human liver gene therapies., AMERICAN SOCIETY OF GENE & CELL THERAPY, 2018-2019
- Development of Experimental Viral Gene Therapies for Glioblastoma., GLIOMA PRECISION MEDICINE PROGRAM, 2018-2019
- Sexually dimorphic viral-host interactions in pediatric liver diseases., STANFORD UNIVERSITY INSTITUTE FOR IMMUNITY, TRANSPLANTATION & INFECTION, 2016-2017
- Novel strategies to expand the adeno-associated virus platform., NIH, 2014-2017
- Characterizing whether clinical and research-grade rAAV vectors differ in post-translational modifications., STANFORD UNIVERSITY MASS SPECTROMETRY CORE FACILITY, 2015-2016
- Determining the Progenitor-Like Capacity of Liver Cancer Cells in vivo., NIH, 2007-2012
Publications (20)
Top publication keywords:
Capsid ProteinsAlkaptonuriaDNA End-Joining RepairDependovirusHydrolasesTransduction, GeneticGenetic VectorsHepatocytesDNA, ViralGene TargetingGenetic EngineeringProtein Processing, Post-TranslationalTyrosinemiasBenzaldehydesGene Expression Regulation, Viral
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Comparison of highly pure rAAV9 vector stocks produced in suspension by PEI transfection or HSV infection reveals striking quantitative and qualitative differences.
Molecular therapy. Methods & clinical development 2021 Trivedi PD, Yu C, Chaudhuri P, Johnson EJ, Caton T, Adamson L, Byrne BJ, Paulk NK, Clément N -
Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics".
Frontiers in immunology 2021 Martinez-Navio JM, Paulk NK, Gao G -
VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector Impurities.
Frontiers in immunology 2021 Rumachik NG, Malaker SA, Paulk NK -
Gene Therapy: It Is Time to Talk about High-Dose AAV.
Genetic Engineering & Biotechnology News 2020 Nicole Paulk -
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors.
Molecular therapy. Methods & clinical development 2020 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
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Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings.
Human gene therapy methods 2019 Maynard LH, Smith O, Tilmans NP, Tham E, Hosseinzadeh S, Tan W, Leenay R, May AP, Paulk NK -
An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging.
Nature chemical biology 2019 Oh Y, Park Y, Cho JH, Wu H, Paulk NK, Liu LX, Kim N, Kay MA, Wu JC, Lin MZ -
Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle.
Molecular therapy. Methods & clinical development 2018 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 -
Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity.
Nature communications 2017 Winters IP, Chiou SH, Paulk NK, McFarland CD, Lalgudi PV, Ma RK, Lisowski L, Connolly AJ, Petrov DA, Kay MA, Winslow MM -
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
Science translational medicine 2017 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, … -
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Molecular therapy : the journal of the American Society of Gene Therapy 2017 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… -
Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.
Nature 2014 Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA -
In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2012 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 -
AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo.
Human gene therapy 2012 Paulk NK, Loza LM, Finegold MJ, Grompe M -
Ochronosis in a murine model of alkaptonuria is synonymous to that in the human condition.
Osteoarthritis and cartilage 2012 Taylor AM, Preston AJ, Paulk NK, Sutherland H, Keenan CM, Wilson PJ, Wlodarski B, Grompe M, Ranganath LR, Gallagher JA, Jarvis JC -
Novel strategies to improve viral gene targeting and therapeutic liver repopulation in vivo
Novel strategies to improve viral gene targeting and therapeutic liver repopulation in vivo 2012 Paulk NK -
Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.
Hepatology (Baltimore, Md.) 2010 Paulk NK, Wursthorn K, Wang Z, Finegold MJ, Kay MA, Grompe M -
Ploidy reductions in murine fusion-derived hepatocytes.
PLoS genetics 2009 Duncan AW, Hickey RD, Paulk NK, Culberson AJ, Olson SB, Finegold MJ, Grompe M -
Effects of DMSO oxidation products on iron speciation in photochemical simulation experiments
Environ Sci Technol 2008 Key J, Paulk N, and Johansen A -
Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice.
Nature biotechnology 2007 Azuma H, Paulk N, Ranade A, Dorrell C, Al-Dhalimy M, Ellis E, Strom S, Kay MA, Finegold M, Grompe M