Ultrasound Microbubbles Enhance Recombinant Adeno-Associated Virus Vector Delivery to Retinal Ganglion Cells In Vivo

Xie, Wenyue; Liu, Su; Su, Hong; Wang, Zhigang; Zheng, Yuanyi; Fu, Yi

doi:10.1016/j.acra.2010.05.008

Cited by 30 publications

(34 citation statements)

References 18 publications

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“…Despite a high number of publications demonstrating the ability of FUS to deliver gene bearing liposomes[152,153], non-viral polyplexes[154–156], viruses[157–159] and free or MB bound plasmid DNA[160] to non-CNS tissues, there are very few studies that have shown the delivery of systemically administered gene-bearing agents to the CNS with FUS. Indeed, the first successful studies showing delivery of reporter gene-bearing agents across the BBB with FUS were completed as recently at 2012[161–163].…”

Section: Gene Deliverymentioning

confidence: 99%

Drug and gene delivery across the blood–brain barrier with focused ultrasound

Timbie

Mead

Price

2015

Journal of Controlled Release

184

129

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The blood-brain barrier (BBB) remains one of the most significant limitations to treatments of central nervous system (CNS) disorders including brain tumors, neurodegenerative diseases and psychiatric disorders. It is now well-established that focused ultrasound (FUS) in conjunction with contrast agent microbubbles may be used to non-invasively and temporarily disrupt the BBB, allowing localized delivery of systemically administered therapeutic agents as large as 100 nm in size to the CNS. Importantly, recent technological advances now permit FUS application through the intact human skull, obviating the need for invasive and risky surgical procedures. When used in combination with magnetic resonance imaging, FUS may be applied precisely to pre-selected CNS targets. Indeed, FUS devices capable of sub-millimeter precision are currently in several clinical trials. FUS mediated BBB disruption has the potential to fundamentally change how CNS diseases are treated, unlocking potential for combinatorial treatments with nanotechnology, markedly increasing the efficacy of existing therapeutics that otherwise do not cross the BBB effectively, and permitting safe repeated treatments. This article comprehensively reviews recent studies on the targeted delivery of therapeutics into the CNS with FUS and offers perspectives on the future of this technology.

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Section: Gene Deliverymentioning

confidence: 99%

Drug and gene delivery across the blood–brain barrier with focused ultrasound

Timbie

Mead

Price

2015

Journal of Controlled Release

184

129

View full text Add to dashboard Cite

show abstract

“…Less apoptotic bodies and no representative DNA fragment were detected in the treatment group. Xie et al [17] reported that UMMD can effectively and safely enhance recombinant adenoassociated virus delivery to RGCs in rats, and it may serve as a novel gene delivery method in gene therapy for glaucomatous optic neuroprotection. Fu et al [18] investigated the protective effect of ultrasound microbubbles mediated transfection of brain-derived neurotrophic factor (BDNF) into the retina and visual cortex on RGCs after optic nerve injury in rats.…”

Section: Discussionmentioning

confidence: 99%

Ultrasound Microbubbles Enhance the Neuroprotective Effect of Mouse Nerve Growth Factor on Intraocular Hypertension-Induced Neuroretina Damage in Rabbits

Shen

Huang

et al. 2016

Journal of Ophthalmology

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Ultrasound microbubble combined optic protection drugs have obvious protective effect on optic nerve damage. This way of targeting drug delivery is becoming more simple, not through the whole body metabolism, avoiding drug via blood circulation when facing the decomposition and the environment in the interference and destruction process of drugs, to maximize the guarantee to reach target organs of drug concentration and to reache the maximum therapeutic effect. The technique of ultrasound microbubbles is safe, controllable, nonimmunogenic, and repeatable. It provides us with a novel idea in the administration of neuroprotective drugs.

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“…The parameters of UTMD used in the present study were taken from various studies (12,13,15,31). Therefore, the application of the UTMD parameters was determined by cell type, to a certain degree.…”

Section: Discussionmentioning

confidence: 99%

Ultrasound-targeted microbubble destruction enhances gene transduction of adeno-associated virus in a less-permissive cell type, NIH/3T3

Jin

Фан

Wang

et al. 2013

Molecular Medicine Reports

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Adeno-associated virus (AAV) is a common vector utilized in gene therapy. The NIH/3T3 cell line, which is a potential induced pluripotent stem (iPS) cell type, was identified to be a less-permissive cell type to AAV due to its defective endosomal processing. Ultrasound-targeted microbubble destruction (UTMD) enhanced the gene transduction of AAV in permissive cells. However, there are no data concerning UTMD enhancement in less-permissive cells, and the exact mechanism of UTMD enhancement in cellular uptake is unclear. Greater knowledge concerning the rate-limiting steps in NIH/3T3 cells would aid in the elucidation of the mechanism of UTMD enhancement in the gene transduction of AAV. In the present study, UTMD enhanced the gene transduction of AAV in NIH/3T3 cells, suggesting that UTMD-enhanced AAV-mediated gene transduction may be beneficial for gene therapy in iPS cells. The dose dependence of UTMD enhancement indicated that mechanisms other than sonoporation were involved in the cellular uptake of AAV. However, UTMD did not greatly increase the gene transduction of AAV in NIH/3T3 cells. Additionally, the similar degree of enhancement in the two cell types resulted in no correlation between UTMD and endosomal processing. Future studies on UTMD-mediated AAV transduction in other non- or less-permissive cell types may aid in elucidating the exact mechanism of UTMD enhancement in cellular uptake.

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Ultrasound Microbubbles Enhance Recombinant Adeno-Associated Virus Vector Delivery to Retinal Ganglion Cells In Vivo

Cited by 30 publications

References 18 publications

Drug and gene delivery across the blood–brain barrier with focused ultrasound

Drug and gene delivery across the blood–brain barrier with focused ultrasound

Ultrasound Microbubbles Enhance the Neuroprotective Effect of Mouse Nerve Growth Factor on Intraocular Hypertension-Induced Neuroretina Damage in Rabbits

Ultrasound-targeted microbubble destruction enhances gene transduction of adeno-associated virus in a less-permissive cell type, NIH/3T3

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