Viral and Nonviral Vectors for In Vivo and Ex Vivo Gene Therapies

Crespo-Barreda, A.; Encabo‐Berzosa, M. Mar; González-Pastor, Rebeca; Ortíz-Teba, P.; Iglesias, María Candela; Serrano, José Luís; Martín-Duque, Pilar

doi:10.1016/b978-0-12-800548-4.00011-5

Cited by 20 publications

(12 citation statements)

References 111 publications

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“…However, this approach lacks target specificity, shows low transfection efficiency and fast degradation. Lipofection was the second most popular non-viral vector and is used in 4.5% of clinical trials worldwide, with cationic lipid/DNA complexes [11]. However, no long-term knock down effect (sustained-release) in cells has been reported for cationic lipids-based gene delivery systems, which means that continuous infusion or frequent repeat administrations are necessary.…”

Section: Introductionmentioning

confidence: 99%

Recent Advances in Chitosan-Based Carriers for Gene Delivery

et al. 2019

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Approximately 4000 diseases are associated with malfunctioning genes in a particular cell type. Gene-based therapy provides a platform to modify the disease-causing genes expression at the cellular level to treat pathological conditions. However, gene delivery is challenging as these therapeutic genes need to overcome several physiological and intracellular barriers in order, to reach the target cells. Over the years, efforts have been dedicated to develop efficient gene delivery vectors to overcome these systemic barriers. Chitosan, a versatile polysaccharide, is an attractive non-viral vector material for gene delivery mainly due to its cationic nature, biodegradability and biocompatibility. The present review discusses the design factors that are critical for efficient gene delivery/transfection and highlights the recent progress of gene therapy using chitosan-based carriers.

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Section: Introductionmentioning

confidence: 99%

Recent Advances in Chitosan-Based Carriers for Gene Delivery

et al. 2019

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“…In addition, cationic lipids and polymers can deliver larger transgenes and can be just as effective as viral methods when used to treat noninherited diseases [26–29]. As of 2012, lipofection is the second most popular nonviral gene modification system in clinical trials with a usage of 5.9% [30]. This study is aimed at finding an effective means of transient transfection that maintains high cell quality by comparing both reported and unreported transfection systems that are commercially available for hBM-MSCs.…”

Section: Introductionmentioning

confidence: 99%

Efficient Nonviral Transfection of Human Bone Marrow Mesenchymal Stromal Cells Shown Using Placental Growth Factor Overexpression

Cheung

Hovey

Gobin

et al. 2018

Stem Cells International

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Background Human mesenchymal stromal/stem cells (hMSCs) hold great therapeutic potential due to their immunomodulatory and tissue regenerative properties. Enhancement of biological features of hMSCs by transfection has become a focus of investigation for cell- and gene-based therapies. However, many of the current transient transfection methods result in either low transfection efficiency or high cytotoxicity. Methods In order to find a transfection method that would address the current issues of low transfection efficiency and high cytotoxicity, 6 commercially available cationic lipid and polymer reagents were tested on human bone marrow-derived MSCs (hBM-MSCs) using GFP as a reporter gene. One transfection method using TransIT-2020 was selected and tested with an emphasis on cell quality (viability, identity, and yield), as well as efficacy with a human placental growth factor (PlGF) plasmid. Results TransIT-2020 yielded the highest fluorescence signal per cell out of the methods that did not decrease cell recovery. Transfecting GFP to 5 hBM-MSC donors using TransIT-2020 yielded 24–36% GFP-expressing cells with a viability of 85–96%. hBM-MSC identity was unaffected as CD90, CD105, and CD73 markers were retained (>95%+) after transfection. When this method was applied to PlGF expression, there was up to a 220-fold increase in secretion. Both growth and secretion of PlGF in overexpressing hBM-MSC were sustained over 7 days, confirming the sustainability and applicability of the TransIT-2020 transfection system. Discussion We report a simple and efficient method for transient transfection that has not been reported for hBM-MSCs, encompassing high levels of plasmid expression without significant changes to fundamental hBM-MSC characteristics.

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“…Viruses that can be modified for their use in gene therapy are adenovirus, herpes simplex virus, vaccinia virus, retrovirus, lentivirus, adeno-associated virus, and reovirus. The transfection efficiency of viruses is superior from other methods, but there are several disadvantages like immune recognition for most of the viruses, mutagenic interaction of retrovirus and lentivirus, and inflammatory toxicity of adenovirus (22,27).…”

Section: Vectors For Dna Transfectionmentioning

confidence: 99%

Nanoparticles for death‑induced gene therapy in cancer (Review)

et al. 2017

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Due to the high toxicity and side effects of the use of traditional chemotherapy in cancer, scientists are working on the development of alternative therapeutic technologies. An example of this is the use of death‑induced gene therapy. This therapy consists of the killing of tumor cells via transfection with plasmid DNA (pDNA) that contains a gene which produces a protein that results in the apoptosis of cancerous cells. The cell death is caused by the direct activation of apoptosis (apoptosis‑induced gene therapy) or by the protein toxic effects (toxin‑induced gene therapy). The introduction of pDNA into the tumor cells has been a challenge for the development of this therapy. The most recent implementation of gene vectors is the use of polymeric or inorganic nanoparticles, which have biological and physicochemical properties (shape, size, surface charge, water interaction and biodegradation rate) that allow them to carry the pDNA into the tumor cell. Furthermore, nanoparticles may be functionalized with specific molecules for the recognition of molecular markers on the surface of tumor cells. The binding between the nanoparticle and the tumor cell induces specific endocytosis, avoiding toxicity in healthy cells. Currently, there are no clinical protocols approved for the use of nanoparticles in death‑induced gene therapy. There are still various challenges in the design of the perfect transfection vector, however nanoparticles have been demonstrated to be a suitable candidate. This review describes the role of nanoparticles used for pDNA transfection and key aspects for their use in death‑induced gene therapy.

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Viral and Nonviral Vectors for In Vivo and Ex Vivo Gene Therapies

Cited by 20 publications

References 111 publications

Recent Advances in Chitosan-Based Carriers for Gene Delivery

Recent Advances in Chitosan-Based Carriers for Gene Delivery

Efficient Nonviral Transfection of Human Bone Marrow Mesenchymal Stromal Cells Shown Using Placental Growth Factor Overexpression

Nanoparticles for death‑induced gene therapy in cancer (Review)

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