Amphoteric Agmatine Containing Polyamidoamines as Carriers for Plasmid DNA In Vitro and In Vivo Delivery

2010 ◽  
Vol 11 (10) ◽  
pp. 2667-2674 ◽  
Author(s):  
Roberta Cavalli ◽  
Agnese Bisazza ◽  
Roberto Sessa ◽  
Luca Primo ◽  
Fabio Fenili ◽  
...  
Keyword(s):  

2017 ◽  
Vol 13 (4) ◽  
pp. 1377-1387 ◽  
Author(s):  
Jayesh A. Kulkarni ◽  
Johnathan Layne Myhre ◽  
Sam Chen ◽  
Yuen Yi C. Tam ◽  
Adrian Danescu ◽  
...  


2006 ◽  
Vol 116 (2) ◽  
pp. e26-e28 ◽  
Author(s):  
F. Tack ◽  
A. Bakker ◽  
S. Maes ◽  
N. Dekeyser ◽  
M. Bruining ◽  
...  


2014 ◽  
Vol 3 ◽  
pp. e212 ◽  
Author(s):  
Suzan M Hammond ◽  
Graham McClorey ◽  
Joel Z Nordin ◽  
Caroline Godfrey ◽  
Sofia Stenler ◽  
...  




2010 ◽  
Vol 47 (3) ◽  
pp. 270-274 ◽  
Author(s):  
Linsey C. Phillips ◽  
Alexander L. Klibanov ◽  
Douglas K. Bowles ◽  
Michael Ragosta ◽  
John A. Hossack ◽  
...  


2018 ◽  
Vol 19 (11) ◽  
pp. 3452 ◽  
Author(s):  
Shingo Nakamura ◽  
Masayuki Ishihara ◽  
Satoshi Watanabe ◽  
Naoko Ando ◽  
Masato Ohtsuka ◽  
...  

Hydrodynamics-based gene delivery (HGD) is an efficient method for transfecting plasmid DNA into hepatocytes in vivo. However, the resulting gene expression is transient, and occurs in a non-tissue specific manner. The piggyBac (PB) transposon system allows chromosomal integration of a transgene in vitro. This study aimed to achieve long-term in vivo expression of a transgene by performing hepatocyte-specific chromosomal integration of the transgene using PB and HGD. Using this approach, we generated a novel mouse model for a hepatic disorder. A distinct signal from the reporter plasmid DNA was discernible in the murine liver approximately two months after the administration of PB transposons carrying a reporter gene. Then, to induce the hepatic disorder, we first administered mice with a PB transposon carrying a CETD unit (loxP-flanked stop cassette, diphtheria toxin-A chain gene, and poly(A) sites), and then with a plasmid expressing the Cre recombinase under the control of a liver-specific promoter. We showed that this system can be used for in situ manipulation and analysis of hepatocyte function in vivo in non-transgenic (Tg) animals.



2021 ◽  
Vol 14 (9) ◽  
pp. 841
Author(s):  
Jian-Ming Lü ◽  
Zhengdong Liang ◽  
Dongliang Liu ◽  
Bin Zhan ◽  
Qizhi Yao ◽  
...  

We previously reported a new polymer, lactic-co-glycolic acid-polyethylenimine (LGA-PEI), as an improved nanoparticle (NP) delivery for therapeutic nucleic acids (TNAs). Here, we further developed two antibody (Ab)-conjugated LGA-PEI NP technologies for active-targeting delivery of TNAs. LGA-PEI was covalently conjugated with a single-chain variable fragment antibody (scFv) against mesothelin (MSLN), a biomarker for pancreatic cancer (PC), or a special Ab fragment crystallizable region-binding peptide (FcBP), which binds to any full Ab (IgG). TNAs used in the current study included tumor suppressor microRNA mimics (miR-198 and miR-520h) and non-coding RNA X-inactive specific transcript (XIST) fragments; green fluorescence protein gene (GFP plasmid DNA) was also used as an example of plasmid DNA. MSLN scFv-LGA-PEI NPs with TNAs significantly improved their binding and internalization in PC cells with high expression of MSLN in vitro and in vivo. Anti-epidermal growth factor receptor (EGFR) monoclonal Ab (Cetuximab) binding to FcBP-LGA-PEI showed active-targeting delivery of TNAs to EGFR-expressing PC cells.



2011 ◽  
Vol 21 ◽  
pp. 230-242 ◽  
Author(s):  
F Wegman ◽  
◽  
A Bijenhof ◽  
L Schuijff ◽  
FC Öner ◽  
...  


2020 ◽  
Author(s):  
Robert L. Kruse ◽  
Xavier Legras ◽  
Mercedes Barzi

AbstractNew therapies against hepatitis B virus (HBV) require the elimination of covalently closed circular DNA (cccDNA), the episomal HBV genome. HBV plasmids containing an overlength 1.3-mer genome and bacterial backbone (pHBV1.3) are used in many different models, but do not replicate the unique features of cccDNA. Since the stable cccDNA pool is a barrier to HBV eradication in patients, we developed a recombinant circular HBV genome (rcccDNA) to mimic the cccDNA using Cre/LoxP technology. We validated four LoxP insertion sites into the HBV genome using hydrodynamic tail vein injection into murine liver, demonstrating high levels of HBV surface antigen (HBsAg) and HBV DNA expression with rcccDNA formation. HBsAg expression from rcccDNA was >30,000 ng/mL over 78 days, while HBsAg-expression from pHBV1.3 plasmid DNA declined from 2,753 ng/mL to 131 ng/mL over that time in immunodeficient mice (P<0.001), reflective of plasmid DNA silencing. We then cloned Cre-recombinase in cis on the LoxP-HBV plasmids, achieving plasmid stability in bacteria with intron insertion into Cre and demonstrating rcccDNA formation after transfection in vitro and in vivo. These cis-Cre/LoxP-HBV plasmids were then used to create HBx-mutant and GFP reporter plasmids to further probe cccDNA biology and antiviral strategies against cccDNA. Overall, we believe these auto-generating rcccDNA plasmids will be of great value to model cccDNA for testing new therapies against HBV infection.





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