Lan Nguyen Weiss
Biography
Lan Nguyen Weiss, MD, PhD, is Assistant Project Scientist IV in the Department of Pediatrics, Division of Genetic and Genomic Medicine, at the University of California, Irvine, where she works under Professor Virginia Kimonis. She holds an MD from Hue University in Vietnam and a PhD in Medical Science in Infection Research from Nagasaki University in Japan, where her doctoral work identified HLA-DRB1*0901 as a protective allele against severe dengue virus infection. Before joining UC Irvine, she served as a researcher and departmental vice head at the Pasteur Institute in Ho Chi Minh City, Vietnam, where she led dengue immunology studies and developed a loop-mediated isothermal amplification diagnostic test for meningitis.
Over more than a decade at UC Irvine, Weiss has built a translational research program focused on rare neuromuscular, lysosomal, and mitochondrial disorders, with particular depth in antisense oligonucleotide therapeutics and AAV-based gene therapy. Her work on glycogen synthase-targeting ASO strategies in Pompe disease and ASO approaches in VCP-associated multisystem proteinopathy has contributed to publications in Clinical and Translational Medicine and to extramural funding including NIH R21AG093390 and a Muscular Dystrophy Association grant. She has also advanced AAV and mRNA-LNP delivery platforms for VCP, NUBPL, and beta-mannosidosis, and has led enzyme replacement therapy evaluation for beta-mannosidosis in disease-relevant cell and mouse models.
Weiss maintains a long-standing collaboration with Ionis Pharmaceuticals on antisense oligonucleotide programs and with JCR Pharmaceuticals Japan on enzyme replacement therapy and CNS drug delivery platform technology. She has presented her research at national and international conferences including the American Society of Gene and Cell Therapy and the Muscular Dystrophy Association annual meeting, and she mentors Bio 199 students, research fellows, and technical staff in the Kimonis laboratory on an ongoing basis.
Return to topEducation
- PhD in Medical Science in Infection Research, Nagasaki University, Japan, 2009
- MD, Hue University, Vietnam, 1995
Distinctions
- Research Fellowship, Pasteur Institute, Paris, France, 1999-2000
- PhD Scholarship, Japanese Ministry of Education, Culture, Sports, Science and Technology, 2004-2009
- NIH Institutional National Research Service Award (NRSA, T32), 2018-2019
- Excellent Paper of the Year, NEKKEN Nagasaki University, 2008
Areas of Expertise
- Rare Neuromuscular Disease Therapeutics
- Antisense Oligonucleotide Drug Development
- Lysosomal Glycogen Pathway Modulation
- iPSC-Derived Muscle Cell Modeling
- AAV Gene Therapy Payload Optimization
- mRNA-LNP CNS Delivery Strategies
- Beta-Mannosidosis Enzyme Replacement
Recent Publications
- Weiss L, Carrer M, Shmara A, Martin A, Yin H, Pal P, Cheng C, Ta L, Boock V, Fazeli Y, Chang M, Paguio M, Lee J, Yu H, Weiss J, Grossman T, Raben N, Jafar-Nejad P, Kimonis V, “Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease”, Clin Transl Med, vol. 15, no. 4, pp. e70314, 2025.
- Pal P, Carrer M, Weiss L, Jaime OG, Cheng C, Shmara A, Boock V, Bosch D, Youssef M, Fazeli Y, Afetian M, Grossman T, Hicks MR, Jafar-Nejad P, Kimonis V, “Antisense oligonucleotides targeting valosin-containing protein ameliorate muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy”, Clin Transl Med, vol. 15, no. 12, pp. e70530, 2025.
- Tedesco B, Vendredy L, Adriaenssens E, Cozzi M, Asselbergh B, Crippa V, Cristofani R, Rusmini P, Ferrari V, Casarotto E, Chierichetti M, Mina F, Pramaggiore P, Galbiati M, Piccolella M, Weiss L, Kimonis V, Timmerman V, Poletti A, “HSPB8 frameshift mutant aggregates weaken chaperone-assisted selective autophagy in neuromyopathies” (opens in new tab), Autophagy, vol. 19, no. 8, pp. 2217-2239, 2023.
- Cheng C, Weiss L, Leinonen H, Shmara A, Yin HZ, Ton T, Do A, Lee J, Ta L, Mohanty E, Vargas J, Weiss J, Palczewski K, Kimonis V, “VCP/p97 inhibitor CB-5083 modulates muscle pathology in a mouse model of VCP inclusion body myopathy” (opens in new tab), Journal of Translational Medicine, vol. 20, no. 1, pp. 21, 2022.
- Cheng C., Cleak J., Weiss L., Cater H., Stewart M., Wells S., Columbres R.C., Shmara A., Morato Torres C.A., Zafar F., Schule B., Neumann J., Hatchwell E., Kimonis V., “Early embryonic lethality in complex I associated p.L104P Nubpl mutant mice” (opens in new tab), Orphanet Journal of Rare Diseases, vol. 17, 2022.
- Bhatnagar C., Shah J., Ramani B., Surampalli A., Avanti M., Radom-Aizik S., Knight M., Weiss L., Caiozzo V., Kimonis V., “Safety and effectiveness of resistance training in patients with late onset Pompe disease - a pilot study” (opens in new tab), Neuromuscular Disorders, vol. 32, pp. 284-294, 2022.
- Weiss L, Jung KM, Nalbandian A, Llewellyn K, Yu H, Ta L, Chang I, Migliore M, Squire E, Ahmed F, Piomelli D, Kimonis V, “Ceramide contributes to pathogenesis and may be targeted for therapy in VCP inclusion body myopathy” (opens in new tab), Human Molecular Genetics, vol. 29, no. 24, pp. 3945-3953, 2021.
- Leinonen H, Cheng C, Pitkänen M, Sander CL, Zhang J, Saeid S, Turunen T, Shmara A, Weiss L, Ta L, Ton T, Koskelainen A, Vargas JD, Kimonis V, Palczewski K, “A p97/valosin-containing protein inhibitor drug CB-5083 has a potent but reversible off-target effect on phosphodiesterase-6” (opens in new tab), Journal of Pharmacology and Experimental Therapeutics, vol. 378, pp. 31-41, 2021.
- Kimonis V., Al Dubaisi R., Maclean A.E., Hall K., Weiss L., Stover A.E., Schwartz P.H., Berg B., Cheng C., Parikh S., Conner B.R., Wu S., Hasso A.N., Scott D.A., Koenig M.K., Karam R., Tang S., Smith M., Chao E., Balk J., et al., “NUBPL mitochondrial disease: New patients and review of the genetic and clinical spectrum” (opens in new tab), Journal of Medical Genetics, vol. 58, pp. 314-325, 2021.
- Cheng C, Weiss L, Ta L, Kimonis V, “Expression level of R155H mRNA in the knock-in mouse model” (opens in new tab), Biochemical and Biophysical Research Communications, vol. 523, no. 4, pp. 985-986, 2020.
- Manh D.H., Weiss L.N., Thuong N.V., Mizukami S., Dumre S.P., Luong Q.C., Thanh L.C., Thang C.M., Huu P.T., Phuc L.H., Nhung C.T.H., Mai N.T., Truong N.Q., Ngu V.T.T., Quoc D.K., Ha T.T.N., Ton T., An T.V., Halhouli O., Quynh L.N., et al., “Kinetics of CD4+ T Helper and CD8+ Effector T Cell Responses in Acute Dengue Patients” (opens in new tab), Frontiers in Immunology, vol. 11, 2020.
- Pare G., Neupane B., Eskandarian S., Harris E., Halstead S., Gresh L., Kuan G., Balmaseda A., Villar L., Rojas E., Osorio J.E., Anh D.D., De Silva A.D., Premawansa S., Premawansa G., Wijewickrama A., Lorenzana I., Parham L., Rodriguez C., Fernandez-Salas I., et al., “Genetic risk for dengue hemorrhagic fever and dengue fever in multiple ancestries” (opens in new tab), Ebiomedicine, vol. 51, 2020.
- Al-Tahan S, Weiss L, Tang S, Saporta M, Mozaffar T, Udd B, Kimonis V, “A new family with HSPB8-associated autosomal dominant rimmed vacuolar myopathy” (opens in new tab), Neurology Genetics, vol. 5, no. 4, pp. e349, 2019.
- Phuong N.T.N., Manh D.H., Dumre S.P., Mizukami S., Weiss L.N., Van Thuong N., Ha T.T.N., Phuc L.H., Van An T., Tieu T.M., Kamel M.G., Morra M.E., Huong V.T.Q., Huy N.T., Hirayama K., “Plasma cell-free DNA: A potential biomarker for early prediction of severe dengue” (opens in new tab), Annals of Clinical Microbiology and Antimicrobials, vol. 18, 2019.
- Mahmoud R., Singh P., Weiss L., Lakatos A., Oakes M., Hossain W., Butler M.G., Kimonis V., “Newborn screening for Prader–Willi syndrome is feasible: Early diagnosis for better outcomes” (opens in new tab), American Journal of Medical Genetics Part A, vol. 179, pp. 29-36, 2019.
- Inokuchi M., Dumre S.P., Mizukami S., Tun M.M.N., Kamel M.G., Manh D.H., Phuc L.H., Van Thuong N., Van An T., Weiss L.N., Turk T., Dang T.N., Huong V.T.Q., Morita K., Huy N.T., Hirayama K., “Association between dengue severity and plasma levels of dengue-specific IgE and chymase” (opens in new tab), Archives of Virology, vol. 163, pp. 2337-2347, 2018.
- Al-Tahan S., Al-Obeidi E., Yoshioka H., Lakatos A., Weiss L., Grafe M., Palmio J., Wicklund M., Harati Y., Omizo M., Udd B., Kimonis V., “Novel valosin-containing protein mutations associated with multisystem proteinopathy” (opens in new tab), Neuromuscular Disorders, vol. 28, pp. 491-501, 2018.
Contact Information
Email: lnweiss@uci.edu
Phone: (949) 824-7964
Address: Hewitt Hall 2501
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Last updated on 9/10/2026.