Paquet, Dominik
Dannert A, Schulz N, Klimmt J, Knez L, Groschup B, Gonçalves CC, Carraro C, Schifferer M, Brendel M, Paquet D. A human iPSC model of tauopathies engineered for 4R tau isoform expression endogenously develops late-stage neuronal tau pathology. Sci Transl Med. 2026 Apr 8;18(844):eadu9845. doi: 10.1126/scitranslmed.adu9845. Epub 2026 Apr 8. PMID: 41950304.
- González-Gallego J, Todorov-Völgyi K, Müller SA, Antesberger S, Todorov MI, Malik R, Grimalt-Mirada R, Gonçalves CC, Schifferer M, Kislinger G, Weisheit I, Lindner B, Crusius D, Kroeger J, Borri M, Erturk A, Nelson M, Misgeld T, Lichtenthaler SF, Dichgans M, Paquet D. A fully iPS-cell-derived 3D model of the human blood-brain barrier for exploring neurovascular disease mechanisms and therapeutic interventions. Nat Neurosci. 2025 Dec 15. doi: 10.1038/s41593-025-02123-w. Epub ahead of print. PMID: 41398476.
Todorov-Völgyi K, González-Gallego J, Müller SA, Todorov MI, Seker FB, Frerich S, Cernilogar FM, Schröger L, Malik R, Cao J, Llovera G, Roth S, Schillinger U, Schifferer M, Reyahi A, Crusius D, Pedro LD, Simons M, Carlsson P, Ertürk A, Liesz A, Schotta G, Plesnila N, Lichtenthaler SF, Paquet D, Dichgans M. The stroke risk gene Foxf2 maintains brain endothelial cell function via Tie2 signaling. Nat Neurosci. 2025 Dec 15. doi: 10.1038/s41593-025-02136-5. Epub ahead of print. PMID: 41398477.
- Carraro C, Montgomery JV, Klimmt J, Paquet D, Schultze JL, Beyer MD. Tackling neurodegeneration in vitro with omics: a path towards new targets and drugs. Front Mol Neurosci. 2024 Jun 17;17:1414886. doi: 10.3389/fnmol.2024.1414886. PMID: 38952421; PMCID: PMC11215216.
- Dinkel L, Hummel S, Zenatti V, Malara M, Tillmann Y, Colombo A, Monasor LS, Suh JH, Logan T, Roth S, Paeger L, Hoffelner P, Bludau O, Schmidt A, Müller SA, Schifferer M, Nuscher B, Njavro JR, Prestel M, Bartos LM, Wind-Mark K, Slemann L, Hoermann L, Kunte ST, Gnörich J, Lindner S, Simons M, Herms J, Paquet D, Lichtenthaler SF, Bartenstein P, Franzmeier N, Liesz A, Grosche A, Bremova-Ertl T, Catarino C, Beblo S, Bergner C, Schneider SA, Strupp M, Di Paolo G, Brendel M, Tahirovic S. Myeloid cell-specific loss of NPC1 in mice recapitulates microgliosis and neurodegeneration in patients with Niemann-Pick type C disease. Sci Transl Med. 2024 Dec 4;16(776):eadl4616. doi: 10.1126/scitranslmed.adl4616. Epub 2024 Dec 4. PMID: 39630885.
- Reich M, Simon MJ, Polke B, Paris I, Werner G, Schrader C, Spieth L, Davis SS, Robinson S, de Melo GL, Schlaphoff L, Buschmann K, Berghoff S, Logan T, Nuscher B, de Weerd L, Edbauer D, Simons M, Suh JH, Sandmann T, Kariolis MS, DeVos SL, Lewcock JW, Paquet D, Capell A, Di Paolo G, Haass C. Peripheral expression of brain-penetrant progranulin rescues pathologies in mouse models of frontotemporal lobar degeneration. Sci Transl Med. 2024 Jun 5;16(750):eadj7308. doi: 10.1126/scitranslmed.adj7308. Epub 2024 Jun 5. PMID: 38838131.
- Titus KR, Simandi Z, Chandrashekar H, Paquet D, Phillips-Cremins JE. Cell-type-specific loops linked to RNA polymerase II elongation in human neural differentiation. Cell Genom. 2024 Jul 9:100606. doi: 10.1016/j.xgen.2024.100606. Epub ahead of print. PMID: 38991604.
- Todorov-Völgyi K, González-Gallego J, Müller SA, Beaufort N, Malik R, Schifferer M, Todorov MI, Crusius D, Robinson S, Schmidt A, Körbelin J, Bareyre F, Ertürk A, Haass C, Simons M, Paquet D, Lichtenthaler SF, Dichgans M. Proteomics of mouse brain endothelium uncovers dysregulation of vesicular transport pathways during aging. Nat Aging. 2024 Mar 22. doi: 10.1038/s43587-024-00598-z. Epub ahead of print. PMID: 38519806.
- Trambauer J, Sarmiento RMR, Garringer HJ, Salbaum K, Pedro LD, Crusius D, Vidal R, Ghetti B, Paquet D, Baumann K, Lindemann L, Steiner H. γ-Secretase modulator resistance of an aggressive Alzheimer-causing presenilin mutant can be overcome in the heterozygous patient state by a set of advanced compounds. Alzheimers Res Ther. 2025 Feb 19;17(1):49. doi: 10.1186/s13195-025-01680-3. PMID: 39972463; PMCID: PMC11837686.
- Dannert A, Klimmt J, Cardoso Gonçalves C, Crusius D, Paquet D (2023). Reproducible and scalable differentiation of highly pure cortical neurons from human induced pluripotent stem cells. STAR Protoc. 2023 May 4;4(2):102266. doi: 10.1016/j.xpro.2023.102266. Epub ahead of print. PMID: 37148244.
- Dawkins E, Derks RJE, Schifferer M, Trambauer J, Winkler E, Simons M, Paquet D, Giera M, Kamp F, Steiner H (2023). Membrane lipid remodeling modulates γ-secretase processivity. J Biol Chem. 2023 Feb 15:103027. doi: 10.1016/j.jbc.2023.103027. Epub ahead of print. PMID: 36805335.
- Gottschlich A, Thomas M, Grünmeier R, Lesch S, Rohrbacher L, Igl V, Briukhovetska D, Benmebarek MR, Vick B, Dede S, Müller K, Xu T, Dhoqina D, Märkl F, Robinson S, Sendelhofert A, Schulz H, Umut Ö, Kavaka V, Tsiverioti CA, Carlini E, Nandi S, Strzalkowski T, Lorenzini T, Stock S, Müller PJ, Dörr J, Seifert M, Cadilha BL, Brabenec R, Röder N, Rataj F, Nüesch M, Modemann F, Wellbrock J, Fiedler W, Kellner C, Beltrán E, Herold T, Paquet D, Jeremias I, von Baumgarten L, Endres S, Subklewe M, Marr C, Kobold S (2023). Single-cell transcriptomic atlas-guided development of CAR-T cells for the treatment of acute myeloid leukemia. Nat Biotechnol. 2023 Mar 13. doi: 10.1038/s41587-023-01684-0. Epub ahead of print. PMID: 36914885.
- Pesämaa I, Müller SA, Robinson S, Darcher A, Paquet D, Zetterberg H, Lichtenthaler SF, Haass C (2023). A microglial activity state biomarker panel differentiates FTD-granulin and Alzheimer's disease patients from controls. Mol Neurodegener. 2023 Sep 29;18(1):70. doi: 10.1186/s13024-023-00657-w. PMID: 37775827; PMCID: PMC10543321.
- Pantazis CB, Yang A, Lara E, McDonough JA, Blauwendraat C, Peng L, Oguro H, Kanaujiya J, Zou J, Sebesta D, Pratt G, Cross E, Blockwick J, Buxton P, Kinner-Bibeau L, Medura C, Tompkins C, Hughes S, Santiana M, Faghri F, Nalls MA, Vitale D, Ballard S, Qi YA, Ramos DM, Anderson KM, Stadler J, Narayan P, Papademetriou J, Reilly L, Nelson MP, Aggarwal S, Rosen LU, Kirwan P, Pisupati V, Coon SL, Scholz SW, Priebe T, Öttl M, Dong J, Meijer M, Janssen LJM, Lourenco VS, van der Kant R, Crusius D, Paquet D, Raulin AC, Bu G, Held A, Wainger BJ, Gabriele RMC, Casey JM, Wray S, Abu-Bonsrah D, Parish CL, Beccari MS, Cleveland DW, Li E, Rose IVL, Kampmann M, Calatayud Aristoy C, Verstreken P, Heinrich L, Chen MY, Schüle B, Dou D, Holzbaur ELF, Zanellati MC, Basundra R, Deshmukh M, Cohen S, Khanna R, Raman M, Nevin ZS, Matia M, Van Lent J, Timmerman V, Conklin BR, Johnson Chase K, Zhang K, Funes S, Bosco DA, Erlebach L, Welzer M, Kronenberg-Versteeg D, Lyu G, Arenas E, Coccia E, Sarrafha L, Ahfeldt T, Marioni JC, Skarnes WC, Cookson MR, Ward ME, Merkle FT (2022). A reference human induced pluripotent stem cell line for large-scale collaborative studies. Cell Stem Cell. 2022 Dec 1;29(12):1685-1702.e22. doi: 10.1016/j.stem.2022.11.004. PMID: 36459969.
- Reifschneider A, Robinson S, van Lengerich B, Gnörich J, Logan T, Heindl S, Vogt MA, Weidinger E, Riedl L, Wind K, Zatcepin A, Pesämaa I, Haberl S, Nuscher B, Kleinberger G, Klimmt J, Götzl JK, Liesz A, Bürger K, Brendel M, Levin J, Diehl-Schmid J, Suh J, Di Paolo G, Lewcock JW, Monroe KM, Paquet D, Capell A, Haass C (2022). Loss of TREM2 rescues hyperactivation of microglia, but not lysosomal deficits and neurotoxicity in models of progranulin deficiency. EMBO J. 2022 Jan 12:e109108. doi: 10.15252/embj.2021109108. Epub ahead of print. PMID: 35019161.
- Scotto Rosato A, Krogsaeter EK, Jaślan D, Abrahamian C, Montefusco S, Soldati C, Spix B, Pizzo MT, Grieco G, Böck J, Wyatt A, Wünkhaus D, Passon M, Stieglitz M, Keller M, Hermey G, Markmann S, Gruber-Schoffnegger D, Cotman S, Johannes L, Crusius D, Boehm U, Wahl-Schott C, Biel M, Bracher F, De Leonibus E, Polishchuk E, Medina DL, Paquet D, Grimm C (2022). TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann-Pick type C1, and Batten disease. EMBO Mol Med. 2022 Aug 5:e15377. doi: 10.15252/emmm.202115377. Epub ahead of print. PMID: 35929194.
- Soldati C, Lopez-Fabuel I, Wanderlingh LG, Garcia-Macia M, Monfregola J, Esposito A, Napolitano G, Guevara-Ferrer M, Scotto Rosato A, Krogsaeter EK, Paquet D, Grimm CM, Montefusco S, Braulke T, Storch S, Mole SE, De Matteis MA, Ballabio A, Sampaio JL, McKay T, Johannes L, Bolaños JP, Medina DL (2021). Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype. EMBO Mol Med. 2021 Aug 19:e13742. doi: 10.15252/emmm.202013742. Epub ahead of print. PMID: 34411438.
- Weisheit I, Kroeger JA, Malik R, Wefers B, Lichtner P, Wurst W, Dichgans M, Paquet D (2021). Simple and reliable detection of CRISPR-induced on-target effects by qgPCR and SNP genotyping. Nat Protoc. 2021 Feb 17. doi: 10.1038/s41596-020-00481-2. Epub ahead of print. PMID: 33597771.
- Klimmt J, Dannert A, Paquet D (2020). Neurodegeneration in a dish: advancing human stem-cell-based models of Alzheimer's disease. Curr Opin Neurobiol. 2020 Feb 26;61:96-104. doi: 10.1016/j.conb.2020.01.008. [Epub ahead of print]
- Weisheit I, Kroeger JA, Malik R, Klimmt J, Crusius D, Dannert A, Dichgans M, Paquet D (2020). Detection of Deleterious On-Target Effects after HDR-Mediated CRISPR Editing. Cell Rep. 2020;31(8):107689. doi:10.1016/j.celrep.2020.107689
- Lafaille FG, Harschnitz O, Lee YS, Zhang P, Hasek ML, Kerner G, Itan Y, Ewaleifoh O, Rapaport F, Carlile TM, Carter-Timofte ME, Paquet D, Dobbs K, Zimmer B, Gao D, Rojas-Duran MF, Kwart D, Rattina V, Ciancanelli MJ, McAlpine JL, Lorenzo L, Boucherit S, Rozenberg F, Halwani R, Henry B, Amenzoui N, Alsum Z, Marques L, Church JA, Al-Muhsen S, Tardieu M, Bousfiha AA, Paludan SR, Mogensen TH, Quintana-Murci L, Tessier-Lavigne M, Smith GA, Notarangelo LD, Studer L, Gilbert W, Abel L, Casanova JL, Zhang SY (2019). Human SNORA31 variations impair cortical neuron-intrinsic immunity to HSV-1 and underlie herpes simplex encephalitis. Nat Med. 2019 Dec;25(12):1873-1884. doi: 10.1038/s41591-019-0672-3. Epub 2019 Dec 5.