Discovery Research in Physiologically Maintained Deceased

Science, 388(6746), pp. 473–476. doi:10.1126/science.adt3527.

Pet, D.B., Parent, B. and Singhal, N.S. (2025) ‘Discovery research in physiologically maintained deceased’

Measurement and clinical interpretation of CRISPR off-targets.

Nat. Genet. https://doi.org/10.1038/s41588-025-02428-3.

Angelini Stewart, A., Ahrens-Nicklas, R.C., Tsai, S.Q., Musunuru, K., Giannikopoulos, P., Clelland, C.D., 2025. Measurement and clinical interpretation of CRISPR off-targets.

Opposing role of phagocytic receptors MERTK and AXL in Progranulin deficient FTD.

Biol. 8, 971. https://doi.org/10.1038/s42003-025-08368-2.

Clelland, C.D., Fan, L., Saloner, R., Etchegaray, J.I., Altobelli, C.R., Salomonsson, S., Maltos, A.M., Sachdev, A., Zhu, J., Lee, S.-I., Li, Y., Zhou, Y., Le, D., Wang, C., Carling, G., Kodama, L., Sayed, F., Perez-Bermejo, J.A., Geier, E.G., Yokoyama, J.S., Rosen, H., Nana, A.L., Spina, S., Grinberg, L.T., Seeley, W.W., Elahi, F., Boxer, A.L., Arkin, M.R., Gan, L., 2025. Opposing role of phagocytic receptors MERTK and AXL in Progranulin deficient FTD. Commun.

nf-core/pacvar: a pipeline for analyzing long-read PacBio whole genome and repeat expansion sequencing data.

Bioinformatics. 2025 Mar 29; 41(4)

Jain T, Clelland C., 2025. nf-core/pacvar: a pipeline for analyzing long-read PacBio whole genome and repeat expansion sequencing data.

Single-Molecule Sequencing of the C9orf72 Repeat Expansion in Patient iPSCs

Bio Protoc 14. https://doi.org/10.21769/BioProtoc.5060.

Tsai, Y.-, Brown, K., Bernardi, M., Harting, J., Clelland, C., 2024. Single-Molecule Sequencing of the C9orf72 Repeat Expansion in Patient iPSCs.

Zfp106 binds to G-quadruplex RNAs and inhibits RAN translation and formation of RNA foci caused by G4C2 repeats.

Proc Natl Acad Sci USA 121, e2220020121. https://doi.org/10.1073/pnas.2220020121.

Celona, B., Salomonsson, S.E., Wu, H., Dang, B., Kratochvil, H.T., Clelland, C.D., DeGrado, W.F., Black, B.L., 2024. Zfp106 binds to G-quadruplex RNAs and inhibits RAN translation and formation of RNA foci caused by G4C2 repeats.

Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excision.

Proc Natl Acad Sci USA 121, e2307814121. https://doi.org/10.1073/pnas.2307814121.

Sachdev, A., Gill, K., Sckaff, M., Birk, A.M., Aladesuyi Arogundade, O., Brown, K.A., Chouhan, R.S., Issagholian-Lewin, P.O., Patel, E., Watry, H.L., Bernardi, M.T., Keough, K.C., Tsai, Y.-C., Smith, A.S.T., Conklin, B.R., Clelland, C.D., 2024. Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excision.

Novel avenues of tau research.

Alzheimers Dement 20, 2240–2261. https://doi.org/10.1002/alz.13533.

Sexton, C.E., Bitan, G., Bowles, K.R., Brys, M., Buée, L., Maina, M.B., Clelland, C.D., Cohen, A.D., Crary, J.F., Dage, J.L., Diaz, K., Frost, B., Gan, L., Goate, A.M., Golbe, L.I., Hansson, O., Karch, C.M., Kolb, H.C., La Joie, R., Lee, S.E., Matallana, D., Miller, B.L., Onyike, C.U., Quiroz, Y.T., Rexach, J.E., Rohrer, J.D., Rommel, A., Sadri-Vakili, G., Schindler, S.E., Schneider, J.A., Sperling, R.A., Teunissen, C.E., Weninger, S.C., Worley, S.L., Zheng, H., Carrillo, M.C., 2024.

Validated assays for the quantification of C9orf72 human pathology.

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Salomonsson, S.E., Maltos, A.M., Gill, K., Aladesuyi Arogundade, O., Brown, K.A., Sachdev, A., Sckaff, M., Lam, K.J.K., Fisher, I.J., Chouhan, R.S., Van Laar, V.S., Marley, C.B., McLaughlin, I., Bankiewicz, K.S., Tsai, Y.C., Conklin, B.R., Clelland, C.D., 2024. Validated assays for the quantification of C9orf72 human pathology.

Building CRISPR gene therapies for the central nervous system: A review.

JAMA Neurol. 81, 283–290. https://doi.org/10.1001/jamaneurol.2023.4983.

Salomonsson, S.E., Clelland, C.D., 2024. Building CRISPR gene therapies for the central nervous system: A review.

Functional characterization of Alzheimer's disease genetic variants in microglia.

Nat. Genet. 55, 1735–1744. https://doi.org/10.1038/s41588-023-01506-8.

Yang, X., Wen, J., Yang, H., Jones, I.R., Zhu, X., Liu, W., Li, B., Clelland, C.D., Luo, W., Wong, M.Y., Ren, X., Cui, X., Song, M., Liu, H., Chen, C., Eng, N., Ravichandran, M., Sun, Y., Lee, D., Van Buren, E., Jiang, M.-Z., Chan, C.S.Y., Ye, C.J., Perera, R.M., Gan, L., Li, Y., Shen, Y., 2023. Functional characterization of Alzheimer’s disease genetic variants in microglia.

Two therapeutic CRISPR/Cas9 gene editing approaches revert FTD/ALS cellular pathology caused by a C9orf72 repeat expansion mutation in patient derived cells

BioRxiv. https://doi.org/10.1101/2022.05.21.492887.

Sckaff, M., Gill, K., Sachdev, A., Birk, A.M., Aladesuyi Arogundade, O., Watry, H.L., Keough, K.C., Tsai, Y.C., Ziegle, J., Conklin, B.R., Clelland, C.D., 2022. Two therapeutic CRISPR/Cas9 gene editing approaches revert FTD/ALS cellular pathology caused by a C9orf72 repeat expansion mutation in patient derived cells.