Localized, non-viral delivery of nucleic acids: Opportunities, challenges and current strategies

Germershaus, Oliver; Nultsch, Kira

doi:10.1016/j.ajps.2014.10.001

Cited by 20 publications

(8 citation statements)

References 129 publications

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“…Além dos vetores citados acima, existem também os vetores lipopoliplexos, que são produzidos unindo-se as propriedades vantajosas dos lipoplexos e dos poliplexos. Os lipopoliplexos são mais estáveis, apresentam maior reprodutibilidade e eficiência de transfecção 16 . Para melhorar a transferência de genes por vetores não virais, como plasmídeos, são utilizadas técnicas de entrega do gene de interesse, como: os métodos químicos chamados de lipossoma catiônico, polímeros; e os métodos físicos, como as técnicas eletromecânicas de eletroporação e de biobalística 8,12 .…”

Section: Vetores Não Viraisunclassified

Vetores virais para uso em terapia gênica

Oliveira

França

Souza

et al. 2018

Revista Pan-Amazônica de Saúde

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RESUMOA terapia gênica tem como princípio a transferência de material genético para dentro das células de um indivíduo, com o intuito de corrigir genes responsáveis por características patológicas e, assim, tratar doenças. Desse modo, a entrega eficiente e a expressão apropriada dos genes terapêuticos são requisitos fundamentais para a eficiência da terapia gênica. Diante disso, os vírus se apresentam como importantes sistemas para a transferência de genes, sendo utilizados como uma excelente estratégia para o tratamento de doenças monogênicas e não monogênicas. Os vetores virais baseados em retrovírus, adenovírus, vírus adenoassociados, herpesvírus e poxvírus têm sido estudados para o tratamento de doenças monogênicas, incluindo a doença granulomatosa crônica, a imunodeficiência combinada grave ligada ao X e a deficiência da enzima adenosina desaminase, assim como para as doenças não monogênicas como o câncer. A presente revisão objetiva explorar os aspectos mais relevantes do uso de vetores virais na terapia gênica. Embora sejam eficientes na entrega do material genético, os vetores virais apresentam risco de infecção, ativam a resposta imune do hospedeiro e podem apresentar um potencial oncogênico. Assim, para que a terapia gênica possa ser aplicada na rotina clínica, muitas pesquisas ainda são necessárias para o refinamento e aperfeiçoamento da técnica, incluindo o desenvolvimento dos vetores virais. ABSTRACTThe gene therapy is designed to introduce genetic material into an individual's cells in order to correct genes responsible for pathological features and thus to treat diseases. Thereby, the efficient delivery and the appropriate expression of therapeutic genes are fundamental requirements for the gene therapy efficiency. Accordingly, the viruses are important systems for genes transference and have been used as an excellent strategy in the treatment of monogenic and non-monogenic diseases. The viral vectors based on retroviruses, adenoviruses, adeno-associated viruses, herpesviruses and poxviruses have been studied for the treatment of monogenic diseases, including chronic granulomatous disease, X-linked severe combined immunodeficiency and adenosine deaminase deficiency, as well as non-monogenic diseases such as cancer. This study aims to explore the most relevant aspects of the use of viral vectors in gene therapy. Although they are effective in delivering genetic material, the viral vectors present risk of infection, activate host immune responses and may present oncogenic potential. Thus, for the gene therapy to be applied in the clinical routine, more studies are necessary to improve the technique, including the development of viral vectors.

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Section: Vetores Não Viraisunclassified

Vetores virais para uso em terapia gênica

Oliveira

França

Souza

et al. 2018

Revista Pan-Amazônica de Saúde

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“…Hydrogels can be formed by crosslinking or by self-assembly from hydrophilic polymers of natural, synthetic, or semi-synthetic origin. The release of encapsulated nucleic acid (NA) can be controlled via the hydrogel pore size, network degradation rate, or through physical interaction or chemical conjugation of the vector system with hydrogel components (Germershaus and Nultsch, 2015).…”

Section: Why Hydrogels?mentioning

confidence: 99%

Engineering approaches in siRNA delivery

Barba

Cascone

Caccavo

et al. 2017

International Journal of Pharmaceutics

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siRNAs are very potent drug molecules, able to silence genes involved in pathologies development. siRNAs have virtually an unlimited therapeutic potential, particularly for the treatment of inflammatory diseases. However, their use in clinical practice is limited because of their unfavorable properties to interact and not to degrade in physiological environments. In particular they are large macromolecules, negatively charged, which undergo rapid degradation by plasmatic enzymes, are subject to fast renal clearance/hepatic sequestration, and can hardly cross cellular membranes. These aspects seriously impair siRNAs as therapeutics. As in all the other fields of science, siRNAs management can be advantaged by physical-mathematical descriptions (modeling) in order to clarify the involved phenomena from the preparative step of dosage systems to the description of drug-body interactions, which allows improving the design of delivery systems/processes/therapies. This review analyzes a few mathematical modeling approaches currently adopted to describe the siRNAs delivery, the main procedures in siRNAs vectors' production processes and siRNAs vectors' release from hydrogels, and the modeling of pharmacokinetics of siRNAs vectors. Furthermore, the use of physical models to study the siRNAs vectors' fate in blood stream and in the tissues is presented. The general view depicts a framework maybe not yet usable in therapeutics, but with promising possibilities for forthcoming applications.

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“…Different NPs have multiple applications in biomedical systems; one of them is gene transformation efficiency relative to the non-viral approaches [25,26]. In mammalian-cultured cells, use of NPs as gene carrier is well known [27][28][29] but their application in plant cells is still limited [30,31].…”

Section: Introductionmentioning

confidence: 99%

Novel nanoplex‐mediated plant transformation approach

Bansod

Bawaskar

Shende

et al. 2019

IET nanobiotechnol.

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Here, a rapid and easy transformation by electroporation technique for gene transfer in plants using cell penetrating amino nanocomplex (nanoplex) has been demonstrated in Nicotiana. Nanoplex was prepared using cell penetrating amino acids (CPAs) such as poly-L-lysine (PLL) and Argenine (Arg), in combination with the gold nanoparticles (AuNPs). PLLsmodified nanoplex with zeta potential of 34.2 ± 1.22 mV charge showed 63.3% efficiency for gene transformation in plant cells as compared to 60% when modified with Arg and the zeta potential was found to be 30.0 ± 0.83 mV; whereas, the transformation efficiency without nanoplex was found to be 6.6%. The findings indicate that the zeta potential of positively charged nanocomplex (AuNPs/CPAs/DNA/CPAs) increases the transformation efficiency because of their ability to protect the DNA from electroporation wave and endogenous enzyme damage. Transformation was confirmed by GUS assay and amplification of npt gene. This technique may open up new possibilities of gene transfer in plants, which will enable to produce large number of transgenic plants.

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Localized, non-viral delivery of nucleic acids: Opportunities, challenges and current strategies

Cited by 20 publications

References 129 publications

Vetores virais para uso em terapia gênica

Vetores virais para uso em terapia gênica

Engineering approaches in siRNA delivery

Novel nanoplex‐mediated plant transformation approach

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