Efficient gene editing via non-viral delivery of CRISPR-Cas9 system using polymeric and hybrid microcarriers
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Efficient gene editing via non-viral delivery of CRISPR-Cas9 system using polymeric and hybrid microcarriers. / Timin, Alexander S; Muslimov, Albert R; Lepik, Kirill V; Epifanovskaya, Olga S; Shakirova, Alena I; Mock, Ulrike; Riecken, Kristoffer; Okilova, Maria V; Sergeev, Vladislav S; Afanasyev, Boris V; Fehse, Boris; Sukhorukov, Gleb B.
in: NANOMED-NANOTECHNOL, Jahrgang 14, Nr. 1, 01.2018, S. 97-108.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Efficient gene editing via non-viral delivery of CRISPR-Cas9 system using polymeric and hybrid microcarriers
AU - Timin, Alexander S
AU - Muslimov, Albert R
AU - Lepik, Kirill V
AU - Epifanovskaya, Olga S
AU - Shakirova, Alena I
AU - Mock, Ulrike
AU - Riecken, Kristoffer
AU - Okilova, Maria V
AU - Sergeev, Vladislav S
AU - Afanasyev, Boris V
AU - Fehse, Boris
AU - Sukhorukov, Gleb B
N1 - Copyright © 2017. Published by Elsevier Inc.
PY - 2018/1
Y1 - 2018/1
N2 - CRISPR-Cas9 is a revolutionary genome-editing technology that has enormous potential for the treatment of genetic diseases. However, the lack of efficient and safe, non-viral delivery systems has hindered its clinical application. Here, we report on the application of polymeric and hybrid microcarriers, made of degradable polymers such as polypeptides and polysaccharides and modified by silica shell, for delivery of all CRISPR-Cas9 components. We found that these microcarriers mediate more efficient transfection than a commercially available liposome-based transfection reagent (>70% vs. <50% for mRNA, >40% vs. 20% for plasmid DNA). For proof-of-concept, we delivered CRISPR-Cas9 components using our capsules to dTomato-expressing HEK293T cells-a model, in which loss of red fluorescence indicates successful gene editing. Notably, transfection of indicator cells translated in high-level dTomato knockout in approx. 70% of transfected cells. In conclusion, we have provided proof-of-principle that our micro-sized containers represent promising non-viral platforms for efficient and safe gene editing.
AB - CRISPR-Cas9 is a revolutionary genome-editing technology that has enormous potential for the treatment of genetic diseases. However, the lack of efficient and safe, non-viral delivery systems has hindered its clinical application. Here, we report on the application of polymeric and hybrid microcarriers, made of degradable polymers such as polypeptides and polysaccharides and modified by silica shell, for delivery of all CRISPR-Cas9 components. We found that these microcarriers mediate more efficient transfection than a commercially available liposome-based transfection reagent (>70% vs. <50% for mRNA, >40% vs. 20% for plasmid DNA). For proof-of-concept, we delivered CRISPR-Cas9 components using our capsules to dTomato-expressing HEK293T cells-a model, in which loss of red fluorescence indicates successful gene editing. Notably, transfection of indicator cells translated in high-level dTomato knockout in approx. 70% of transfected cells. In conclusion, we have provided proof-of-principle that our micro-sized containers represent promising non-viral platforms for efficient and safe gene editing.
KW - Journal Article
U2 - 10.1016/j.nano.2017.09.001
DO - 10.1016/j.nano.2017.09.001
M3 - SCORING: Journal article
C2 - 28917642
VL - 14
SP - 97
EP - 108
JO - NANOMED-NANOTECHNOL
JF - NANOMED-NANOTECHNOL
SN - 1549-9634
IS - 1
ER -
