Nicole Paulk • UCSF Profiles

Education and Training

Stanford University	Postdoc	08/2016	Human Gene Therapy
Oregon Health & Science University	PhD	06/2012	Viral Gene Therapy
Central Washington University	BS	06/2006	Medical Microbiology

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.

Interests

AAV, Gene Therapy, Bioengineering, Cancer, Rare Disease, Immunotherapy, Technology Development, Proteomics, Genomics, Epigenomics, Sexual Dimorphism

Websites

	Paulk Lab Addgene Plasmids
	Paulk Lab GitHub Code
	Paulk Lab Protocols.io
	Nicole Paulk ORCID
	Nicole Paulk Google Scholar
	Nicole Paulk LinkedIn
	Nicole Paulk ResearchGate

In The News

	Quote in Gene & Engineering News on Audentes XLMTM trial (09/15/2021)
	Quote in Biopharma Dive on XLMTM gene therapy (09/14/2021)
	Podcast with Phacilitate UK on the FDA AAV AdComm (09/12/2021)
	Quote in Endpoints News on FDA Adcomm (09/03/2021)
	Quote in Phacilitate on FDA Adcomm (08/30/2021)
	STAT Panel on "Gene Therapy's Next Steps" (08/12/2021)
	ASGCT Writing Recommendations (06/18/2021)
	Interview on the future of AAV gene therapy (05/17/2021)
	Quote in Endpoints News on liver biomarkers during AAV treatment (04/14/2021)
	Interview on Next-Gen AAV Therapies (03/12/2021)
	Quote in STAT News on differences between lentivirus and AAV (02/17/2021)
	Endpoints Webinar on "Beyond AAV 1.0" (01/28/2021)
	Quote in Endpoints News on gene therapy's future (01/26/2021)
	Featured in the Thermo E-Book on AAV Manufacturing Complexities (12/10/2020)
	Breakthrough in 'The Vector' by ASGCT on our AAV proteomics study (12/10/2020)
	Quote in Gene & Engineering News on gene therapy's renaissance (10/02/2020)
	Webiner with Gene & Engineering News on AAV gene therapy (08/12/2020)
	Interview with Gene & Engineering News on high dose AAV (07/07/2020)
	Interview with Bioproduction Summit on gene therapy manufacturing challenges (04/19/2020)
	Interview with American Society of Gene & Cell Therapy on women in gene therapy (03/26/2020)
	OpEd in Cell & Gene on "Why complex disease needs gene therapy" (03/03/2020)
	Interview with Gene & Engineering News on the future of gene therapy (01/03/2020)
	Quote in MIT Tech Review on California's new biohacking law for at-home genome editing (08/09/2019)
	Interview with C&EN on the buzz in gene therapy (05/02/2019)
	Podcast with Genialis on the importance of precision medicine in academic research (04/30/2019)
	Interview with The Lab Manager on how to make your science dollars go further (05/06/2018)
	Interview with The Verge about the lack of appropriate scientist stock photos on Google image search (05/03/2018)

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.
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 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 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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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.

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.

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.

View in: PubMed Mentions: Fields:
All Allergy and Immunology
Gene Therapy: It Is Time to Talk about High-Dose AAV. Genetic Engineering & Biotechnology News. 2020 Sep 1; 40(9):14-16. Nicole Paulk. .

View in: Publisher Site Mentions:
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.

View in: PubMed Mentions:
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.

View in: PubMed Mentions: 5 Fields:
Gen Genetics The Therapeutics
Translation:Humans Animals Cells
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.

View in: PubMed Mentions: 5 Fields:
Bio Biology Che Chemistry
Translation:Animals
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.

View in: PubMed Mentions:
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.

View in: PubMed Mentions: 23 Fields:
Bio Biology Sci Science
Translation:Animals Cells
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid a-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.

View in: PubMed Mentions: 38 Fields:
Med Medicine (General)Sci Science
Translation:Animals Cells
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.

View in: PubMed Mentions: 47 Fields:
Mol Molecular Biology The Therapeutics
Translation:Humans Animals Cells
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.

View in: PubMed Mentions: 98 Fields:
Sci Science
Translation:Humans Animals Cells
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.

View in: PubMed Mentions: 7 Fields:
Mol Molecular Biology The Therapeutics
Translation:Animals Cells
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.

View in: PubMed Mentions: 15 Fields:
Gen Genetics The Therapeutics
Translation:Animals Cells
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.

View in: PubMed Mentions: 14 Fields:
Ort Orthopedics Rhe Rheumatology
Translation:Animals Cells
Novel strategies to improve viral gene targeting and therapeutic liver repopulation in vivo. 2012. Paulk NK. . View Publication.
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.

View in: PubMed Mentions: 47 Fields:
Gas Gastroenterology
Translation:Animals Cells
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.

View in: PubMed Mentions: 39 Fields:
Gen Genetics
Translation:Animals Cells
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.
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.

View in: PubMed Mentions: 283 Fields:
Bio Biotechnology
Translation:Humans Animals Cells

Nicole Paulk, PhD