Ocular gene delivery systems using ternary complexes of plasmid DNA, polyethylenimine, and anionic polymers.

Ocular gene delivery systems using ternary complexes of plasmid DNA, polyethylenimine, and anionic polymers. Biol Pharm Bull. 2013;36(1):96-101 Authors: Kurosaki T, Uematsu M, Shimoda K, Suzuma K, Nakai M, Nakamura T, Kitahara T, Kitaoka T, Sasaki H Abstract In this experiment, we developed anionic ternary complexes for effective and safe ocular gene delivery. Ternary complexes were constructed by coating plasmid DNA (pDNA)/polyethylenimine (PEI) complex with anionic polymers such as γ-polyglutamic acid (γ-PGA) and chondroitin sulfate (CS). The cationic pDNA/PEI complex showed high gene expression on the human retinal pigment epithelial cell line, ARPE-19 cells. The pDNA/PEI complexes, however, also showed high cytotoxicity on the cells and aggregated strongly in the vitreous body. On the other hand, the anionic ternary complexes showed high gene expression on ARPE-19 cells without such cytotoxicity and aggregation. After intravitreous administration of the complexes, the anionic ternary complexes showed high gene expression in the retina. These results strongly indicate that anionic ternary complexes are suitable for effective and safe ocular gene therapy. PMID: 23302641 [PubMed - in process]
Source: Biological and Pharmaceutical Bulletin - Category: Drugs & Pharmacology Authors: Tags: Biol Pharm Bull Source Type: research

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Conclusion: The review identified the need for tailored educational activities, and in particular the importance of addressing misconceptions. There is also a need for robust qualitative research considering perspectives on current and forthcoming licensed therapies.PMID:34553606 | DOI:10.2217/rme-2020-0181
Source: Regenerative Medicine - Category: Genetics & Stem Cells Authors: Source Type: research
This article will illustrate the low risk of immune response to AAV serotype 2 vector-mediated gene therapy to the brain with support from clinical trial data in aromatic L-amino acid decarboxylase deficiency and Parkinson disease.PMID:34551695 | DOI:10.2174/1566523221666210922155413
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Conclusion: The review identified the need for tailored educational activities, and in particular the importance of addressing misconceptions. There is also a need for robust qualitative research considering perspectives on current and forthcoming licensed therapies.PMID:34553606 | DOI:10.2217/rme-2020-0181
Source: Regenerative Medicine - Category: Genetics & Stem Cells Authors: Source Type: research
This article will illustrate the low risk of immune response to AAV serotype 2 vector-mediated gene therapy to the brain with support from clinical trial data in aromatic L-amino acid decarboxylase deficiency and Parkinson disease.PMID:34551695 | DOI:10.2174/1566523221666210922155413
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