Early entry and deformation of macropinosomes correlates with high efficiency of decaarginine-polyethylene glycol-lipid-mediated gene delivery

2012 ◽  
Vol 14 (4) ◽  
pp. 262-271 ◽  
Author(s):  
Shouhei Kobayashi ◽  
Yoshiyuki Hattori ◽  
Hiroko Osakada ◽  
Kazunori Toma ◽  
Yoshie Maitani ◽  
...  
2017 ◽  
Vol 4 (10) ◽  
pp. 170822 ◽  
Author(s):  
Liping Wu ◽  
Jinshan Xie ◽  
Tan Li ◽  
Zihao Mai ◽  
Lu Wang ◽  
...  

We recently developed a polyethylenimine (PEI) and polyethylene glycol (PEG) dual-functionalized reduced graphene oxide (GO) (PEG−nrGO−PEI, RGPP) for high-efficient gene delivery in HepG2 and Hela cell lines. To evaluate the feasibility and applicability of RGPP as a gene delivery carrier, we here assessed the transfection efficiency of RGPP on gene plasmids and siRNA in 11 different cell lines. Commercial polyalkyleneimine cation transfection reagent (TR) was used as comparison. In HepG2 cells, RGPP exhibited much stronger delivery ability for siRNA and large size plasmids than TR. For green fluorescent protein (GFP) plasmid, RGPP showed about 47.1% of transfection efficiency in primary rabbit articular chondrocytes, and about 27% of transfection efficiency in both SH-SY5Y and A549 cell lines. RGPP exhibited about 37.2% of GFP plasmid transfection efficiency in EMT6 cells and about 26.0% of GFP plasmid transfection efficiency in LO2 cells, but induced about 33% of cytotoxicity in both cell lines. In 4T1 and H9C2 cell lines, RGPP had less than 10% of GFP plasmid transfection efficiency. Collectively, RGPP is a potential nano-carrier for high-efficiency gene delivery, and needs to be further optimized for different cell lines.


2020 ◽  
Vol 20 ◽  
Author(s):  
L. Hajba ◽  
A. Guttman

: Adeno-associated virus (AAV) is one of the most promising viral gene delivery vectors with long-term gene expression and disease correction featuring high efficiency and excellent safety in human clinical trials. During the production of AAV vectors,there are several quality control (QC)parameters that should be rigorously monitored to comply with clini-cal safety and efficacy. This review gives a short summary of the most frequently used AVV production and purification methods,focusing on the analytical techniques applied to determine the full/empty capsid ratio and the integrity of the encapsidated therapeutic DNA of the products.


2002 ◽  
Vol 4 (2) ◽  
pp. 161-169 ◽  
Author(s):  
Elisabetta Ravot ◽  
Giuditta Comolli ◽  
Franco Lori ◽  
Julianna Lisziewicz

2014 ◽  
Vol 22 (7) ◽  
pp. 757-764 ◽  
Author(s):  
Tae-Hun Kim ◽  
Ho Won Seo ◽  
Jin Han ◽  
Kyung Soo Ko ◽  
Joon Sig Choi

2021 ◽  
Author(s):  
Tudor Vasiliu ◽  
Bogdan Florin Florin Craciun ◽  
Andrei Neamtu ◽  
Lilia Clima ◽  
Dragos Lucian Isac ◽  
...  

The biocompatible hydrophilic polyethylene glycol (PEG) is widely used in biomedical applications, such as drug or gene delivery, tissue engineering or as antifouling in biomedical devices. Experimental studies have shown...


2001 ◽  
Vol 37 (7) ◽  
pp. 1975-1980 ◽  
Author(s):  
Philippe G Amabile ◽  
Jacob M Waugh ◽  
Thomas N Lewis ◽  
Christopher J Elkins ◽  
Wolfgang Janas ◽  
...  

Circulation ◽  
2020 ◽  
Vol 142 (Suppl_3) ◽  
Author(s):  
Keerat Kaur ◽  
Asharee Mahmoud ◽  
Hanna Girard ◽  
Ann Anu Kurian ◽  
Magdalena Zak ◽  
...  

Introduction: Despite various clinical modalities, ischemic heart disease remains among the leading causes of mortality and morbidity worldwide. The elemental problem is the immense loss of cardiomyocytes (CMs) post-myocardial infarction (MI). Reprogramming non- cardiomyocytes (non-CMs) into cardiomyocyte (CM)-like cells in vivo is a promising strategy for cardiac regeneration: however, the traditional viral delivery method hampered its application into clinical settings due to low and erratic transduction efficiency. Methods: We used a modified mRNA (modRNA) gene delivery platform to deliver different stoichiometry of cardiac-reprogramming genes (Gata4, Mef2c, Tbx5 and Hand2) together with reprogramming helper genes (Dominant Negative (DN)-TGFβ, DN- Wnt8a and Acid ceramidase (AC)), named 7G, to induce direct cardiac reprogramming post myocardial infarction (MI). Results: Here, we identified 7G modRNA cocktail as an important regulator ofthe cardiac reprogramming. Cardiac transfection with 7G modRNA doubled cardiac reprogramming efficiency (57%) in comparison to Gata4, Mef2C and Tbx5 (GMT) alone (28%) in vitro . By inducing MI in our lineage tracing model, we showed that one-time delivery of the 7G-modRNA cocktail reprogrammed ~25% of the non-CMs in the scar area to CM- like cells. Furthermore, 7G modRNA treated mice showed significantly improved cardiac function, longer survival, reduced scar size and greater capillary density than control mice 28 days post-MI. We attributed the improvement in heart function post modRNA delivery of 7G or 7G with increased Hand2 ratio (7G-GMT Hx2) to significant upregulation of 15 key angiogenic factors without any signs of angioma or edema. Conclusions: 7G or 7G GMT HX2 modRNA cocktails boosts de novo CM-like cells and promotes cardiovascular regeneration post-MI. Thus, we highlight that this gene delivery approach not only has high efficiency but also high margin of safety for translation to clinics.


2012 ◽  
Vol 101A (7) ◽  
pp. 1857-1864 ◽  
Author(s):  
Kumar Singarapu ◽  
Ivy Pal ◽  
Joshua D. Ramsey

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