Author

C. Nicolas

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Bioengineering· 1 Cell Biology· 1

Signature Assays, Organisms & Methods

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Assay:ATAC-seq· 1 Research Focus:cell type annotation· 1 Research Focus:clustering· 1 Research Focus:differential expression· 1 Approach:experimental assay· 1 Research Focus:gene regulatory network inference· 1 Approach:genetic perturbation / CRISPR· 1 Organism:human· 1

Frequent Co-Authors

A. Bhagwate· 1 shared B. T. Dejene· 1 shared C. Lee· 1 shared C. Sindhu· 1 shared C. Van Lith· 1 shared D. O'Brien· 1 shared E. J. Kildebeck· 1 shared G. Bao· 1 shared

Activity by Year

2018-2021 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 21 mutual co-author links

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1 shared paperCell Biology

A. Bhagwate

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Author profile →

2 papers

Recent·Most cited

In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

C. Nicolas, C. Van Lith, K. Allen, R. Hickey, Z. Du, L. Hillin, R. Guthman, W. Cao, A. Bhagwate, D. O'Brien, J.-P. Kocher, R. A. Kaiser, S. Russell, J. Lillegard

This study cures hereditary tyrosinemia type 1 in pigs using in vivo lentiviral gene therapy, preventing liver fibrosis and tumorigenic pathways.

bioRxiv · Jan 2, 2021Cell Biology

Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells

M. Pavel-Dinu, V. Wiebking, B. T. Dejene, W. Srifa, S. Mantri, C. Nicolas, C. Lee, G. Bao, E. J. Kildebeck, N. Punjya, C. Sindhu, M. A. Inlay, N. Saxena, S. S. DeRavin, H. Malech, , M. Porteus

This study corrects SCID-X1 in hematopoietic stem cells using CRISPR/Cas9, achieving high integration frequencies and low off-target effects, supporting clinical development.

bioRxiv · Aug 22, 2018Bioengineering