Modified cyclodextrins as novel non-viral vectors for neuronal siRNA delivery: focus on Huntington’s disease Author(s) Godinho, Bruno M. D. C. Publication date 2014 Original citation Godinho, B. M. D. C. 2014. Modified cyclodextrins as novel non-viral vectors for neuronal siRNA delivery: focus on Huntington’s disease. PhD Thesis, University College Cork. Type of publication Doctoral thesis Righ...

Objective(s): Although viral vectors are considered efficient gene transfer agents, their board application has been limited by toxicity, immunogenicity, mutagenicity and small gene carrying capacity. Non-viral vectors are safe but they suffer from low transfection efficiency. In the present study, polyallylamine (PAA) in two molecular weights (15 and 65 kDa) was modified by alkane derivatives ...

The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (CRISPR/Cas9) genome editing system has revolutionized the field of for therapeutic applications genetic diseases and cancers, development disease models, identification targets drug development. However, safe efficient delivery CRISPR/Cas9 in vivo remains a significant challenge. Compared to vira...

Viral vectors have a wide variety of applications ranging from fundamental studies of viruses to therapeutics. Recombinant viral vectors are usually constructed using methods of reverse genetics to obtain the genetic material of the viral vector. The physicochemical properties of DNA and RNA make them unable to access cells by themselves, and they require assistance to achieve intracellular del...

The host immune responses, including T lymphocytes mediated immune response and humoral immune responses are the important parts of the challenges in gene therapy. There are some potential immunostimulants in gene delivery systems, such as viral and non-viral vectors. Viral gene products, transgene products, viral proteins derived from viral particles required by dead-end infection, and CpG DNA...

DNA based transposon vectors offer a mechanism for non-viral gene delivery into mamma‐ lian and human cells. These vectors work via a cut-and-paste mechanim whereby transpo‐ son DNA containing a transgene(s) of interest is integrated into chromosomal DNA by a transposase enzyme. The first DNA based transposon system which worked efficienty in human cells was sleeping beauty. This was followed a...