Highly efficient genome editing of human hematopoietic stem cells via a nano-silicon-blade delivery approach

Integrative Biology : Quantitative Biosciences From Nano to Macro
Yuan MaLidong Qin

Abstract

Recently, the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 bacterial immunity system has opened a promising avenue to treat genetic diseases that affect the human hematopoietic stem cells (HSCs). Therefore, finding a highly efficient delivery method capable of modifying the genome in the hard-to-transfect HSCs, combined with the advanced CRISPR-Cas9 system, may meet the challenges for dissecting the hematologic disease mechanisms and facilitate future clinical applications. Here, we developed an effective HSC-specified delivery microfluidic chip to disrupt the cell membrane transiently by inducing rapid mechanical deformation that allowed the delivery of biomaterials into the cytoplasm from the surrounding matrix. Compared with the previous designs, the new nano-silicon-blade structure was specifically optimized for HSCs. Using the silicon substrate, the sharpness and rigidity of the nano-blade constriction was largely enhanced to improve the biomaterials delivery efficiency. We achieved highly efficient delivery results by transporting various macro-molecules into the HSCs. Moreover, the treated HSCs possess high viability and maintain inherent pluripotency after the delivery via the Nano-Blade Chip ...Continue Reading

References

Mar 17, 2004·Proceedings of the National Academy of Sciences of the United States of America·Paul Wiggins, Rob Phillips
May 4, 2004·Nature Reviews. Cancer·Claus Nerlov
Dec 3, 2004·The New England Journal of Medicine·Matthew L SmithJude Fitzgibbon
Mar 22, 2006·Nature Immunology·Gregor B Adams, David T Scadden
Apr 28, 2006·The New England Journal of Medicine·Edward A Copelan
Nov 15, 2008·Analytical Chemistry·Jun WangChang Lu
Feb 2, 2010·Journal of Controlled Release : Official Journal of the Controlled Release Society·Tao GengChang Lu
Apr 21, 2012·Journal of Controlled Release : Official Journal of the Controlled Release Society·Yihong ZhanChang Lu
Jan 5, 2013·Science·Le CongFeng Zhang
Jan 24, 2013·Proceedings of the National Academy of Sciences of the United States of America·Armon ShareiKlavs F Jensen
Aug 14, 2013·Proceedings of the National Academy of Sciences of the United States of America·Zhonggang HouJames A Thomson
May 30, 2014·Nature·Pietro GenoveseLuigi Naldini
May 25, 2015·Journal of Biotechnology·Xiquan LiangJonathan D Chesnut
Jun 13, 2015·Human Gene Therapy·Jennifer L GoriDavid Bumcrot
Jul 29, 2015·Proceedings of the National Academy of Sciences of the United States of America·On Shun PakHoward A Stone
Nov 26, 2015·Science Advances·Xin HanLidong Qin
Mar 2, 2016·Proceedings of the National Academy of Sciences of the United States of America·Ming WangQiaobing Xu

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Citations

Feb 18, 2021·Advanced Materials·Dorsa Morshedi RadMajid Ebrahimi Warkiani
Jun 8, 2021·Advanced Science·Jeongsoo Hur, Aram J Chung
Jul 17, 2021·Advanced Materials·Pengchao ZhangLidong Qin
Jul 28, 2018·Chemical Reviews·Martin P StewartKlavs F Jensen
Sep 18, 2020·ACS Applied Materials & Interfaces·Jason N BellingPaul S Weiss

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