Gene and Cell Therapy

Nóbrega, Clévio; Mendonça, Liliana; Matos, Carlos A.

doi:10.1007/978-3-030-41333-0_1

Cited by 5 publications

(4 citation statements)

References 33 publications

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“…Cellular uptake and early endosome escape are the main factors affecting effective gene effectively delivery ( Nóbrega et al, 2020 ). Motivated by efficient VEGFA silencing, cell uptake was determined by flow cytometry and microscope.…”

Section: Resultsmentioning

confidence: 99%

Efficient ocular delivery of siRNA via pH-sensitive vehicles for corneal neovascularization inhibition

Cao

Wang

Deng

et al. 2023

International Journal of Pharmaceutics: X

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Section: Resultsmentioning

confidence: 99%

Efficient ocular delivery of siRNA via pH-sensitive vehicles for corneal neovascularization inhibition

Cao

Wang

Deng

et al. 2023

International Journal of Pharmaceutics: X

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“…The therapeutic target determines the administration routes and gene delivery system when designing a gene therapy. [ 49 ] Gene therapy can be delivered to the affected individual either via in vivo or ex vivo therapy. [ 50 ] Vector-based in vivo therapy offers direct delivery of the therapeutic gene.…”

Section: Current State Of Therapeutics In Paediatric Genetic Diseasesmentioning

confidence: 99%

“…[ 50 ] On the other hand, cell-based ex vivo therapy is a technically challenging multi-step process because it requires a dedicated cell therapy facility for gene modification on the cells, and these modified cells then need to be transplanted into the individuals. [ 49 ] Most of the Food and Drug Administration (FDA)-approved in vivo gene therapies use viral vectors, rather than non-viral systems, for their highly efficient ability to transduce cells with the therapeutic gene sequence. The selection of an ideal vector is dependent on the expected effect, size of the transgene and the safety profile.…”

Section: Current State Of Therapeutics In Paediatric Genetic Diseasesmentioning

confidence: 99%

Therapeutics in paediatric genetic diseases: current and future landscape

Koh

Jamuar

2023

Singapore Medical Journal

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There are more than 7,000 paediatric genetic diseases (PGDs) but less than 5% have treatment options. Treatment strategies targeting different levels of the biological process of the disease have led to optimal health outcomes in a subset of patients with PGDs, where treatment is available. In the past 3 decades, there has been rapid advancement in the development of novel therapies, including gene therapy, for many PGDs. The therapeutic success of treatment relies heavily on knowledge of the genetic basis and the disease mechanism. Specifically, gene therapy has been shown to be effective in various clinical trials, and indeed, these trials have led to regulatory approvals, paving the way for gene therapies for other types of PGDs. In this review, we provide an overview of the treatment strategies and focus on some of the recent advancements in therapeutics for PGDs.

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“…Unspecific molecular interactions, endothelial barriers, and the immune response are the three main extracellular barriers. Limitations of cellular uptake, endosome–lysosome escape, intracellular trafficking, and nuclear delivery are intracellular kinds of barriers [ 8 ]. So, the correct choice of a specific vector for gene delivery is an effective parameter for the success of gene therapy.…”

Section: Introductionmentioning

confidence: 99%

Carbon-Based Nanostructures as Emerging Materials for Gene Delivery Applications

Yazdani,

Mozaffarian,

Pazuki

et al. 2024

Pharmaceutics

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Gene therapeutics are promising for treating diseases at the genetic level, with some already validated for clinical use. Recently, nanostructures have emerged for the targeted delivery of genetic material. Nanomaterials, exhibiting advantageous properties such as a high surface-to-volume ratio, biocompatibility, facile functionalization, substantial loading capacity, and tunable physicochemical characteristics, are recognized as non-viral vectors in gene therapy applications. Despite progress, current non-viral vectors exhibit notably low gene delivery efficiency. Progress in nanotechnology is essential to overcome extracellular and intracellular barriers in gene delivery. Specific nanostructures such as carbon nanotubes (CNTs), carbon quantum dots (CQDs), nanodiamonds (NDs), and similar carbon-based structures can accommodate diverse genetic materials such as plasmid DNA (pDNA), messenger RNA (mRNA), small interference RNA (siRNA), micro RNA (miRNA), and antisense oligonucleotides (AONs). To address challenges such as high toxicity and low transfection efficiency, advancements in the features of carbon-based nanostructures (CBNs) are imperative. This overview delves into three types of CBNs employed as vectors in drug/gene delivery systems, encompassing their synthesis methods, properties, and biomedical applications. Ultimately, we present insights into the opportunities and challenges within the captivating realm of gene delivery using CBNs.

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Gene and Cell Therapy

Cited by 5 publications

References 33 publications

Efficient ocular delivery of siRNA via pH-sensitive vehicles for corneal neovascularization inhibition

Efficient ocular delivery of siRNA via pH-sensitive vehicles for corneal neovascularization inhibition

Therapeutics in paediatric genetic diseases: current and future landscape

Carbon-Based Nanostructures as Emerging Materials for Gene Delivery Applications

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