A Brief Introduction to Current Cancer Gene Therapy

Kobelt, Dennis; Pahle, Jessica; Walther, Wolfgang

doi:10.1007/978-1-0716-2441-8_1

Cited by 7 publications

(5 citation statements)

References 148 publications

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“…Early gene therapy primarily involved the introduction of a therapeutic gene to repair or replace a defective gene [ 235 ]. With the emergence and development of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (CRISPR/Cas9) technology, efficient gene knockout, site-specific gene insertion, and whole-genome screening have become achievable [ 236 ].…”

Section: Nim Delivery Systems Based On Different Therapeutic Strategi...mentioning

confidence: 99%

Multifunctional nano-in-micro delivery systems for targeted therapy in fundus neovascularization diseases

Liu,

Huang,

Zhang

et al. 2024

J Nanobiotechnol

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Fundus neovascularization diseases are a series of blinding eye diseases that seriously impair vision worldwide. Currently, the means of treating these diseases in clinical practice are continuously evolving and have rapidly revolutionized treatment opinions. However, key issues such as inadequate treatment effectiveness, high rates of recurrence, and poor patient compliance still need to be urgently addressed. Multifunctional nanomedicine can specifically respond to both endogenous and exogenous microenvironments, effectively deliver drugs to specific targets and participate in activities such as biological imaging and the detection of small molecules. Nano-in-micro (NIM) delivery systems such as metal, metal oxide and up-conversion nanoparticles (NPs), quantum dots, and carbon materials, have shown certain advantages in overcoming the presence of physiological barriers within the eyeball and are widely used in the treatment of ophthalmic diseases. Few studies, however, have evaluated the efficacy of NIM delivery systems in treating fundus neovascular diseases (FNDs). The present study describes the main clinical treatment strategies and the adverse events associated with the treatment of FNDs with NIM delivery systems and summarizes the anatomical obstacles that must be overcome. In this review, we wish to highlight the principle of intraocular microenvironment normalization, aiming to provide a more rational approach for designing new NIM delivery systems to treat specific FNDs. Graphical Abstract

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Section: Nim Delivery Systems Based On Different Therapeutic Strategi...mentioning

confidence: 99%

Multifunctional nano-in-micro delivery systems for targeted therapy in fundus neovascularization diseases

Liu,

Huang,

Zhang

et al. 2024

J Nanobiotechnol

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show abstract

“…With the objective to deliver nucleic acids such as RNA [ 31 , 32 ], DNA [ 33 , 34 ] or antisense oligonucleotides [ 35 , 36 ] to cure human diseases, gene therapy has attracted great attention in the past several decades. Since the first gene medicine (Vitravene) approved by FDA in 1998, 40 gene therapy products have been officially approved by drug regulatory agencies worldwide.…”

Section: Cd-based Polyrotaxanesmentioning

confidence: 99%

Cyclodextrin-Based Polymeric Drug Delivery Systems for Cancer Therapy

Liu

Qiu

2023

Polymers

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Cyclodextrins (CDs) are one of the most extensively studied cyclic-oligosaccharides due to their low toxicity, good biodegradability and biocompatibility, facile chemical modification, and unique inclusion capacity. However, problems such as poor pharmacokinetics, plasma membrane disruption, hemolytic effects and a lack of target specificity still exist for their applications as drug carriers. Recently, polymers have been introduced into CDs to combine the advantages of both biomaterials for the superior delivery of anticancer agents in cancer treatment. In this review, we summarize four types of CD-based polymeric carriers for the delivery of chemotherapeutics or gene agents for cancer therapy. These CD-based polymers were classified based on their structural properties. Most of the CD-based polymers were amphiphilic with the introduction of hydrophobic/hydrophilic segments and were able to form nanoassemblies. Anticancer drugs could be included in the cavity of CDs, encapsulated in the nanoparticles or conjugated on the CD-based polymers. In addition, the unique structures of CDs enable the functionalization of targeting agents and stimuli-responsive materials to realize the targeting and precise release of anticancer agents. In summary, CD-based polymers are attractive carriers for anticancer agents.

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“…Nucleic acid sequences can be rapidly engineered to silence, augment, or replace defective genes in the body. [1][2][3][4][5][6][7] Accordingly, the usage of nucleic acids as drugs has emerged as an attractive strategy to prevent and treat disease. [8][9][10][11][12][13][14][15] Despite their therapeutic potential, many nucleic acids are unable to act therapeutically unless they can enter target cells in the body.…”

Section: Introductionmentioning

confidence: 99%

Tannic Acid Lipid Nanoparticles can Deliver Messenger RNA Payloads and Improve their Endosomal Escape

Fenton

2023

Advanced Therapeutics

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Even though lipid nanoparticles (LNPs) can deliver messenger RNA (mRNA) payloads into cells, their efficiency is often limited by endosomal trapping which prevents RNA payloads from acting therapeutically. Improving the percentage of RNA LNPs that can escape from endosomes and enter the cytoplasm is therefore an area of active research interest that could lead to improved safety profiles and reduced manufacturing costs of mRNA drugs. Here, tannic acid mRNA LNPs [TA(+) mRNA LNPs] are reported as an effective delivery platform for the delivery of mRNA payloads. The formulation, characterization, and stability of TA(+) mRNA LNPs are described; two different approaches via confocal microscopy are then utilized to quantify the endosomal escape of the TA(+) mRNA LNPs; and lastly, the biodistribution and tolerability of the TA(+) mRNA LNPs are evaluated in mice following intravenous and intramuscular dosing regimens. To isolate the effect that TA imparts on each of these properties, mRNA LNPs that do not contain TA [TA(−) mRNA LNPs] are evaluated side‐by‐side in each of these studies. A collective analysis of these results suggests that TA(+) mRNA LNPs are effective carriers for mRNA payloads and that the incorporation of TA within each formulation improves the endosomal escape of mRNA LNPs.

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A Brief Introduction to Current Cancer Gene Therapy

Cited by 7 publications

References 148 publications

Multifunctional nano-in-micro delivery systems for targeted therapy in fundus neovascularization diseases

Multifunctional nano-in-micro delivery systems for targeted therapy in fundus neovascularization diseases

Cyclodextrin-Based Polymeric Drug Delivery Systems for Cancer Therapy

Tannic Acid Lipid Nanoparticles can Deliver Messenger RNA Payloads and Improve their Endosomal Escape

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