Carsten Charlesworth
Postdoctoral Scholar

Carsten Charlesworth, Ph.D. is a Postdoctoral Scholar in the laboratory of Professor Hiromitsu Nakauchi at Stanford University. Originally from Australia, he earned his undergraduate degree in Biology from Humboldt State University and later joined Stanford through the CIRM Bridges program, working in Dr. Matthew Porteus’s lab on genome editing in hematopoietic stem cells (HSCs) for the treatment of genetic blood disorders. He went on to complete his Ph.D. in Stanford’s Stem Cell and Regenerative Medicine program.

Carsten’s research bridges genome engineering, stem cell biology, and immunology. His work has helped shape the field’s understanding of the immune challenges facing gene therapies. In a Nature Medicine study, he and colleagues identified widespread preexisting immunity to Cas9 proteins in humans, highlighting a major consideration for the clinical use of CRISPR technologies.

At the Nakauchi lab, Carsten is leading efforts to develop new in vivo gene delivery strategies, including SPIT (Secreted Particle Information Transfer) and AAV6-based approaches for HSCs. His work aims to make genome editing safer, more efficient, and clinically translatable.

 

Selected Publications:

• Charlesworth CT, Deshpande PS, Dever DP, Camarena J, Lemgart VT, Cromer MK, Vakulskas CA, Collingwood MA, Zhang L, Bode NM, Behlke MA, Dejene B, Cieniewicz B, Romano R, Lesch BJ, Gomez-Ospina N, Mantri S, Pavel-Dinu M, Weinberg KI, Porteus MH. Identification of preexisting adaptive immunity to Cas9 proteins in humans. Nat Med. 2019 Feb;25(2):249-254. doi: 10.1038/s41591-018-0326-x. Epub 2019 Jan 28. PMID: 30692695; PMCID: PMC7199589.

• Charlesworth CT, Homma S, Suchy F, Wang S, Bhadhury J, Amaya AK, Camarena J, Zhang J, Tan TK, Igarishi K, Nakauchi H. Secreted Particle Information Transfer (SPIT) - A Cellular Platform for In Vivo Genetic Engineering. bioRxiv [Preprint]. 2024 Jan 12:2024.01.11.575257. doi: 10.1101/2024.01.11.575257. PMID: 38260654; PMCID: PMC10802600.

• Charlesworth CT, Camarena J, Cromer MK, Vaidyanathan S, Bak RO, Carte JM, Potter J, Dever DP, Porteus MH. Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting. Mol Ther Nucleic Acids. 2018 Sep 7;12:89-104. doi: 10.1016/j.omtn.2018.04.017. Epub 2018 May 3. PMID: 30195800; PMCID: PMC6023838.

• Charlesworth CT, Homma S, Amaya AK, Dib C, Vaidyanathan S, Tan TK, Miyauchi M, Nakauchi Y, Suchy FP, Wang S, Igarashi KJ, Cromer MK, Dudek AM, Amorin A, Czechowicz A, Wilkinson AC, Nakauchi H. Highly efficient in vivo hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virus. Mol Ther Methods Clin Dev. 2025 Feb 21;33(1):101438. doi: 10.1016/j.omtm.2025.101438. PMID: 40129926; PMCID: PMC11930595.

• Toral MA, Charlesworth CT, Ng B, Chemudupati T, Homma S, Nakauchi H, Bassuk AG, Porteus MH, Mahajan VB. Investigation of Cas9 antibodies in the human eye. Nat Commun. 2022 Feb 25;13(1):1053. doi: 10.1038/s41467-022-28674-1. Erratum in: Nat Commun. 2022 Apr 12;13(1):2109. doi: 10.1038/s41467-022-29844-x. PMID: 35217666; PMCID: PMC8881612.