Microbubbles and Nanobubbles with Ultrasound for Systemic Gene Delivery

Endo‐Takahashi, Yoko; Negishi, Yoichi

doi:10.3390/pharmaceutics12100964

Cited by 71 publications

(70 citation statements)

References 94 publications

(112 reference statements)

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“…Ultrasound targeted MBs destruction (UTMD) is an emerging method for delivering target genes into cells or living animal organs. It has become a research hotspot due to its advantages of simplicity, non-invasiveness, targeting and reproducibility ( 37 – 39 ). Low-frequency ultrasound has little effect on the biological interaction between healthy cells and normal tissue and is safe.…”

Section: Discussionmentioning

confidence: 99%

CMBs carrying PTX and CRISPR/Cas9 targeting C‑erbB‑2 plasmids interfere with endometrial cancer cells

2021

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Development of combination therapy to decrease side effects of chemotherapeutic drugs and increase their utilization rate in combination with gene editing is a key research topic in tumor treatment. The present study aimed to investigate the effect of cationic microbubbles (CMBs) carrying paclitaxel (PTX) and C-erbB-2 knockout plasmid on the endometrial cancer cell line HEC-1A and to determine how C-erbB-2 regulates the function of endometrial cancer cells. Cells were treated with CMB, PTX, PTX-CMBs, cationic plasmid-carrying or cationic PTX-carrying plasmid groups. After verifying the most effective combination of PTX-CMBs and plasmids, HEC-1A cells were transfected. Reverse transcription-quantitative (RT-q)PCR and western blotting were used to measure C-erbB-2 and protein expression. After verifying C-erbB-2 knockout, invasion, healing, clone formation and proliferation of HEC-1A cells were assessed. Simultaneously, expression levels of the genes for P21, P27 , mammalian target of rapamycin ( mTOR ), and Bcl-2 associated death promoter ( Bad ) were measured by RT-qPCR. Compared with the PTX group, CMBs significantly enhanced the absorption efficiency of PTX by HEC-1A cells. C-erbB-2 knockout had an inhibitory effect on the proliferation, migration and invasion of HEC-1A cells; cell proliferation and invasion of the group carrying PTX and plasmids simultaneously were significantly weakened. The C-erbB-2 -knockout group exhibited increased expression of P21 and P27 . Simultaneously loading PTX and plasmid may be novel combination therapy with great potential. C-erbB-2 may regulate the proliferation of HEC-1A cells by downregulating expression of P21 and P27 .

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Section: Discussionmentioning

confidence: 99%

CMBs carrying PTX and CRISPR/Cas9 targeting C‑erbB‑2 plasmids interfere with endometrial cancer cells

2021

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“…FUS irradiation of a targeted region of the brain and the presence of MBs in the blood cause the transient disruption of the BBB, leading to promoting the permeation of nanoparticle through the BBB. Several reports demonstrated that FUS and MBs can be utilized to deliver therapeutic genes for curing neurodegenerative disease, especially in a PD mouse model [ 264 , 265 , 266 , 267 , 268 , 269 ]. There is strong evidence to show that neurotrophic factor can promote the regeneration of dopaminergic neurons to releive the PD syndrome [ 270 , 271 , 272 ].…”

Section: Non-viral Brain Targeting By Non-invasive Methodsmentioning

confidence: 99%

Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB

Kimura

Harashima

2020

Pharmaceutics

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The era of the aging society has arrived, and this is accompanied by an increase in the absolute numbers of patients with neurological disorders, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD). Such neurological disorders are serious costly diseases that have a significant impact on society, both globally and socially. Gene therapy has great promise for the treatment of neurological disorders, but only a few gene therapy drugs are currently available. Delivery to the brain is the biggest hurdle in developing new drugs for the central nervous system (CNS) diseases and this is especially true in the case of gene delivery. Nanotechnologies such as viral and non-viral vectors allow efficient brain-targeted gene delivery systems to be created. The purpose of this review is to provide a comprehensive review of the current status of the development of successful drug delivery to the CNS for the treatment of CNS-related disorders especially by gene therapy. We mainly address three aspects of this situation: (1) blood-brain barrier (BBB) functions; (2) adeno-associated viral (AAV) vectors, currently the most advanced gene delivery vector; (3) non-viral brain targeting by non-invasive methods.

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“…22 Transcutaneous ultrasound has been proven to be efficient for the delivery of genetic material to the liver of mice and pigs. 23,24 So far, the use of these approaches in liver-directed gene therapy is still restricted to experimental animal studies.…”

Section: Administration Of Naked Genetic Materialsmentioning

confidence: 99%

Novel vectors and approaches for gene therapy in liver diseases

Maestro

Weber²,

Zabaleta

et al. 2021

JHEP Reports

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This is a PDF file of an article that has undergone enhancements after acceptance, such as the addition of a cover page and metadata, and formatting for readability, but it is not yet the definitive version of record. This version will undergo additional copyediting, typesetting and review before it is published in its final form, but we are providing this version to give early visibility of the article. Please note that, during the production process, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain.

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Microbubbles and Nanobubbles with Ultrasound for Systemic Gene Delivery

Cited by 71 publications

References 94 publications

CMBs carrying PTX and CRISPR/Cas9 targeting C‑erbB‑2 plasmids interfere with endometrial cancer cells

CMBs carrying PTX and CRISPR/Cas9 targeting C‑erbB‑2 plasmids interfere with endometrial cancer cells

Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB

Novel vectors and approaches for gene therapy in liver diseases

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