Advances in Targeted Gene Delivery

Begum, Anjuman Ara; Tóth, István; Hussein, Waleed M.; Moyle, Peter M.

doi:10.2174/1567201816666190529072914

Cited by 18 publications

(8 citation statements)

References 169 publications

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“…Liposomes and lipid nanoparticles protect nucleic acids from degradation and improve pharmacokinetics [ 1 ], forming the basis for mRNA vaccines against COVID-19. Recombinant viral vectors, including both lentivirus [ 2 ] and adeno-associated virus (AAV) [ 3 ], provide high transfection levels to effectively deliver mRNA and DNA to specific target organs [ 4 ]. In addition, nanoparticles can be used to treat solid tumors, e.g., through the generation of hydroxyl radicals that inhibit tumor growth [ 5 ].…”

Section: Introductionmentioning

confidence: 99%

Retention and Fouling during Nanoparticle Filtration: Implications for Membrane Purification of Biotherapeutics

et al. 2022

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One major challenge in the development of nanoparticle-based therapeutics, including viral vectors for the delivery of gene therapies, is the development of cost-effective purification technologies. The objective of this study was to examine fouling and retention behaviors during the filtration of model nanoparticles through membranes of different pore sizes and the effect of solution conditions. Data were obtained with 30 nm fluorescently labeled polystyrene latex nanoparticles using both cellulosic and polyethersulfone membranes at a constant filtrate flux, and both pressure and nanoparticle transmission were evaluated as a function of cumulative filtrate volume. The addition of NaCl caused a delay in nanoparticle transmission and an increase in fouling. Nanoparticle transmission was also a function of particle hydrophobicity. These results provide important insights into the factors controlling transmission and fouling during nanoparticle filtration as well as a framework for the development of membrane processes for the purification of nanoparticle-based therapeutics.

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Section: Introductionmentioning

confidence: 99%

Retention and Fouling during Nanoparticle Filtration: Implications for Membrane Purification of Biotherapeutics

et al. 2022

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“…These carriers can merge with cell membranes and insert the gene codes into cells. They induce temporary expression of target genes, last a few days, and disappear in dividing cells after a short time [122,123]. Also, these vectors are inexpensive and have low immunogenicity, but they cannot integrate transgenes into the genome, and they just induce a temporary expression of desired genes [17].…”

Section: Transduction Methods For Car-nk Cell Engineeringmentioning

confidence: 99%

Renaissance of armored immune effector cells, CAR-NK cells, brings the higher hope for successful cancer therapy

et al. 2021

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In recent decades, a new method of cellular immunotherapy was introduced based on engineering and empowering the immune effector cells. In this type of immunotherapy, the immune effector cells are equipped with chimeric antigen receptor (CAR) to specifically target cancer cells. In much of the trials and experiments, CAR-modified T cell immunotherapy has achieved very promising therapeutic results in the treatment of some types of cancers and infectious diseases. However, there are also some considerable drawbacks in the clinical application of CAR-T cells although much effort is in progress to rectify the issues. In some conditions, CAR-T cells initiate over-activated and strong immune responses, therefore, causing unexpected side-effects such as systemic cytokine toxicity (i.e., cytokine release syndrome), neurotoxicity, on-target, off-tumor toxicity, and graft-versus-host disease (GvHD). To overcome these limitations in CAR-T cell immunotherapy, NK cells as an alternative source of immune effector cells have been utilized for CAR-engineering. Natural killer cells are key players of the innate immune system that can destroy virus-infected cells, tumor cells, or other aberrant cells with their efficient recognizing capability. Compared to T cells, CAR-transduced NK cells (CAR-NK) have several advantages, such as safety in clinical use, non-MHC-restricted recognition of tumor cells, and renewable and easy cell sources for their preparation. In this review, we will discuss the recent preclinical and clinical studies, different sources of NK cells, transduction methods, possible limitations and challenges, and clinical considerations.

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“…Although viral vectors remain by far the most popular delivery strategy, nonviral vectors are becoming more common. [ 28 , 29 , 30 , 31 ]…”

Section: Gene Therapymentioning

confidence: 99%

Recent Advances in Stimulus‐Responsive Nanocarriers for Gene Therapy

Cheng

et al. 2021

Advanced Science

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Gene therapy provides a promising strategy for curing monogenetic disorders and complex diseases. However, there are challenges associated with the use of viral delivery vectors. The advent of nanomedicine represents a quantum leap in the application of gene therapy. Recent advances in stimulus-responsive nonviral nanocarriers indicate that they are efficient delivery systems for loading and unloading of therapeutic nucleic acids. Some nanocarriers are responsive to cues derived from the internal environment, such as changes in pH, redox potential, enzyme activity, reactive oxygen species, adenosine triphosphate, and hypoxia. Others are responsive to external stimulations, including temperature gradients, light irradiation, ultrasonic energy, and magnetic field. Multiple stimuli-responsive strategies have also been investigated recently for experimental gene therapy.

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Advances in Targeted Gene Delivery

Cited by 18 publications

References 169 publications

Retention and Fouling during Nanoparticle Filtration: Implications for Membrane Purification of Biotherapeutics

Retention and Fouling during Nanoparticle Filtration: Implications for Membrane Purification of Biotherapeutics

Renaissance of armored immune effector cells, CAR-NK cells, brings the higher hope for successful cancer therapy

Recent Advances in Stimulus‐Responsive Nanocarriers for Gene Therapy

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