Home Newsletters Cell Therapy News Improving the Safety of Human Pluripotent Stem Cell Therapies Using Genome-Edited Orthogonal...

Improving the Safety of Human Pluripotent Stem Cell Therapies Using Genome-Edited Orthogonal Safeguards

0
Scientists used genome editing to engineer a general platform to improve the safety of future human pluripotent stem cell-derived cell transplantation therapies.
[Nature Communications]
6445212 {1254621:636P8JXC} apa 50 1 150604 https://www.stemcellsciencenews.com/wp-content/plugins/zotpress/
%7B%22status%22%3A%22success%22%2C%22updateneeded%22%3Afalse%2C%22instance%22%3A%22zotpress-beb7bdab19fa32f87963740ffdb24dd1%22%2C%22meta%22%3A%7B%22request_last%22%3A0%2C%22request_next%22%3A0%2C%22used_cache%22%3Atrue%7D%2C%22data%22%3A%5B%7B%22key%22%3A%22636P8JXC%22%2C%22library%22%3A%7B%22id%22%3A1254621%7D%2C%22meta%22%3A%7B%22creatorSummary%22%3A%22Martin%20et%20al.%22%2C%22parsedDate%22%3A%222020-06-01%22%2C%22numChildren%22%3A2%7D%2C%22bib%22%3A%22%3Cdiv%20class%3D%5C%22csl-bib-body%5C%22%20style%3D%5C%22line-height%3A%202%3B%20padding-left%3A%201em%3B%20text-indent%3A-1em%3B%5C%22%3E%5Cn%20%20%3Cdiv%20class%3D%5C%22csl-entry%5C%22%3EMartin%2C%20R.%20M.%2C%20Fowler%2C%20J.%20L.%2C%20Cromer%2C%20M.%20K.%2C%20Lesch%2C%20B.%20J.%2C%20Ponce%2C%20E.%2C%20Uchida%2C%20N.%2C%20Nishimura%2C%20T.%2C%20Porteus%2C%20M.%20H.%2C%20%26amp%3B%20Loh%2C%20K.%20M.%20%282020%29.%20Improving%20the%20safety%20of%20human%20pluripotent%20stem%20cell%20therapies%20using%20genome-edited%20orthogonal%20safeguards.%20%3Ci%3ENature%20Communications%3C%5C%2Fi%3E%2C%20%3Ci%3E11%3C%5C%2Fi%3E%281%29%2C%202713.%20%3Ca%20href%3D%27https%3A%5C%2F%5C%2Fdoi.org%5C%2F10.1038%5C%2Fs41467-020-16455-7%27%3Ehttps%3A%5C%2F%5C%2Fdoi.org%5C%2F10.1038%5C%2Fs41467-020-16455-7%3C%5C%2Fa%3E%20%3Ca%20title%3D%27Cite%20in%20RIS%20Format%27%20class%3D%27zp-CiteRIS%27%20href%3D%27https%3A%5C%2F%5C%2Fwww.stemcellsciencenews.com%5C%2Fwp-content%5C%2Fplugins%5C%2Fzotpress%5C%2Flib%5C%2Frequest%5C%2Frequest.cite.php%3Fapi_user_id%3D1254621%26amp%3Bitem_key%3D636P8JXC%27%3ECite%3C%5C%2Fa%3E%20%3C%5C%2Fdiv%3E%5Cn%3C%5C%2Fdiv%3E%22%2C%22data%22%3A%7B%22itemType%22%3A%22journalArticle%22%2C%22title%22%3A%22Improving%20the%20safety%20of%20human%20pluripotent%20stem%20cell%20therapies%20using%20genome-edited%20orthogonal%20safeguards%22%2C%22creators%22%3A%5B%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Renata%20M.%22%2C%22lastName%22%3A%22Martin%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Jonas%20L.%22%2C%22lastName%22%3A%22Fowler%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22M.%20Kyle%22%2C%22lastName%22%3A%22Cromer%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Benjamin%20J.%22%2C%22lastName%22%3A%22Lesch%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Ezequiel%22%2C%22lastName%22%3A%22Ponce%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Nobuko%22%2C%22lastName%22%3A%22Uchida%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Toshinobu%22%2C%22lastName%22%3A%22Nishimura%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Matthew%20H.%22%2C%22lastName%22%3A%22Porteus%22%7D%2C%7B%22creatorType%22%3A%22author%22%2C%22firstName%22%3A%22Kyle%20M.%22%2C%22lastName%22%3A%22Loh%22%7D%5D%2C%22abstractNote%22%3A%22Despite%20their%20rapidly-expanding%20therapeutic%20potential%2C%20human%20pluripotent%20stem%20cell%20%28hPSC%29-derived%20cell%20therapies%20continue%20to%20have%20serious%20safety%20risks.%20Transplantation%20of%20hPSC-derived%20cell%20populations%20into%20preclinical%20models%20has%20generated%20teratomas%20%28tumors%20arising%20from%20undifferentiated%20hPSCs%29%2C%20unwanted%20tissues%2C%20and%20other%20types%20of%20adverse%20events.%20Mitigating%20these%20risks%20is%20important%20to%20increase%20the%20safety%20of%20such%20therapies.%20Here%20we%20use%20genome%20editing%20to%20engineer%20a%20general%20platform%20to%20improve%20the%20safety%20of%20future%20hPSC-derived%20cell%20transplantation%20therapies.%20Specifically%2C%20we%20develop%20hPSC%20lines%20bearing%20two%20drug-inducible%20safeguards%2C%20which%20have%20distinct%20functionalities%20and%20address%20separate%20safety%20concerns.%20In%20vitro%20administration%20of%20one%20small%20molecule%20depletes%20undifferentiated%20hPSCs%20%3E106-fold%2C%20thus%20preventing%20teratoma%20formation%20in%20vivo.%20Administration%20of%20a%20second%20small%20molecule%20kills%20all%20hPSC-derived%20cell-types%2C%20thus%20providing%20an%20option%20to%20eliminate%20the%20entire%20hPSC-derived%20cell%20product%20in%20vivo%20if%20adverse%20events%20arise.%20These%20orthogonal%20safety%20switches%20address%20major%20safety%20concerns%20with%20pluripotent%20cell-derived%20therapies.%22%2C%22date%22%3A%222020-06-01%22%2C%22language%22%3A%22en%22%2C%22DOI%22%3A%2210.1038%5C%2Fs41467-020-16455-7%22%2C%22ISSN%22%3A%222041-1723%22%2C%22url%22%3A%22https%3A%5C%2F%5C%2Fwww.nature.com%5C%2Farticles%5C%2Fs41467-020-16455-7%22%2C%22deleted%22%3A1%2C%22collections%22%3A%5B%5D%2C%22dateModified%22%3A%222020-06-09T19%3A51%3A12Z%22%7D%7D%5D%7D
Martin, R. M., Fowler, J. L., Cromer, M. K., Lesch, B. J., Ponce, E., Uchida, N., Nishimura, T., Porteus, M. H., & Loh, K. M. (2020). Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards. Nature Communications, 11(1), 2713. https://doi.org/10.1038/s41467-020-16455-7 Cite
Full Article
Exit mobile version