Lipid nanoparticles loaded with ribonucleoprotein–oligonucleotide complexes synthesized using a microfluidic device exhibit robust genome editing and hepatitis B virus inhibition

Suzuki, Yuichi; Onuma, Haruno; Sato, Rei; Sato, Yusuke; Hashiba, Akari; Maeki, Masatoshi; Tokeshi, Manabu; Kayesh, Mohammad Enamul Hoque; Kohara, Michinori; Tsukiyama-Kohara, Kyoko; Harashima, Hideyoshi

doi:10.1016/j.jconrel.2020.12.013

Cited by 70 publications

(58 citation statements)

References 53 publications

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“…An alternative approach is the use of nanoparticles. Although confirmation in the clinical setting is still required, such nanoparticulated drugs (nanodrugs) have recently gained recognition in drug delivery for a number of therapeutic areas [ 118 , 119 , 120 ]. Indeed, Geiger et al showed that nanoparticles carrying IGF-1 successfully penetrated bovine cartilage within 2 days of application [ 121 ].…”

Section: Optimisation Of Oligonucleotide Methods Of Delivery Into Joint Tissuesmentioning

confidence: 99%

Oligonucleotide Therapies in the Treatment of Arthritis: A Narrative Review

2021

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Osteoarthritis (OA) and rheumatoid arthritis (RA) are two of the most common chronic inflammatory joint diseases, for which there remains a great clinical need to develop safer and more efficacious pharmacological treatments. The pathology of both OA and RA involves multiple tissues within the joint, including the synovial joint lining and the bone, as well as the articular cartilage in OA. In this review, we discuss the potential for the development of oligonucleotide therapies for these disorders by examining the evidence that oligonucleotides can modulate the key cellular pathways that drive the pathology of the inflammatory diseased joint pathology, as well as evidence in preclinical in vivo models that oligonucleotides can modify disease progression.

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Section: Optimisation Of Oligonucleotide Methods Of Delivery Into Joint Tissuesmentioning

confidence: 99%

Oligonucleotide Therapies in the Treatment of Arthritis: A Narrative Review

2021

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“…Type BmNPV: (Chen et al, 2017), (Dong et al, 2018a), (Dong et al, 2018b), (Dong et al, 2019a), (Dong et al, 2019b) Hepatitis B virus: , (Kostyusheva et al, 2019), (Li et al, 2018), (Liu et al, 2018), (Schiwon et al, 2018), (Stone et al, 2021), (Suzuki et al, 2021), (Yan et al, 2021), (Yang et al, 2020) HIV-1: (Bella et al, 2018), (Dash et al, 2019), (Ebina et al, 2013), (Hu et al, 2014), (Kaminski et al, 2016a), (Kaminski et al, 2016b) Finally, we note that RNA interference (RNAi) for silencing viral gene expression is a possible alternative therapy. RNAi, like Cas13, also offers a sequence-targeted antiviral approach in this case by binding to and degrading specific RNA transcripts (Levanova and Poranen, 2018).…”

Section: Targetmentioning

confidence: 97%

The Application of CRISPR/Cas Systems for Antiviral Therapy

Baddeley

Isalan

2021

Front. Genome Ed.

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As CRISPR/Cas systems have been refined over time, there has been an effort to apply them to real world problems, such as developing sequence-targeted antiviral therapies. Viruses pose a major threat to humans and new tools are urgently needed to combat these rapidly mutating pathogens. Importantly, a variety of CRISPR systems have the potential to directly cleave DNA and RNA viral genomes, in a targeted and easily-adaptable manner, thus preventing or treating infections. This perspective article highlights recent studies using different Cas effectors against various RNA viruses causing acute infections in humans; a latent virus (HIV-1); a chronic virus (hepatitis B); and viruses infecting livestock and animal species of industrial importance. The outlook and remaining challenges are discussed, particularly in the context of tacking newly emerging viruses, such as SARS-CoV-2.

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“…Amongst nonviral delivery systems, lipid nanoparticles (LNPs) are clearly the most advanced. There are ample publications highlighting their efficacy and safety when it comes to the delivery of genetic cargo in general [5,23] and CRISPR [24][25][26] specifically. Yet, although LNPs efficiently encapsulate mRNA, there are challenges when it comes to the loading of RNP due to the bulkiness of the Cas9 protein and challenges when it comes to loading.…”

Section: Current Gene Delivery Systemsmentioning

confidence: 99%

The Delivery Challenge of Genome Editing in Human Epithelia

Calderón

Hedtrich

2021

Adv Healthcare Materials

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Despite exciting advances in gene editing, their clinical translation is still hampered by the lack of delivery systems that can encapsulate and deliver gene editing tools like CRISPR-Cas9 or prime editors to the target side. This is particularly challenging in human epithelia, such as the skin and the lung; the latter of which being a mucosal surface that is covered by a mucus layer. In this perspective, the design and biological assessment of delivery systems for gene editing tools like CRISPR in skin and mucosal surfaces are discussed. The current state-of-the-art, current knowledge, and translational gaps, and guide toward improved translation are highlighted. Biological Barriers to Gene Editing in Human EpitheliaGene therapies, including RNA-based approaches, gene augmentation, and gene editing, are powerful tools to prevent, treat, and cure a plethora of human diseases. The first gene therapy, Glybera, obtained regulatory approval in 2012 and at least eight more followed since then. Currently, the drug development pipelines and clinical trials are full of gene therapy-based approaches that hold high hopes for the treatment of previously untreatable conditions. The discovery of CRISPR-based gene editing in 2012 significantly accelerated the genome editing field and resulted in the Nobel Prize just eight years later. [1] CRISPR-Cas is a twoor three-component system that is composed of a target-specific guide RNA (gRNA), a Cas9 nuclease, and in certain cases a donor template. The gRNA complexed nuclease is directed to the DNA target site where it induces double-strand breaks (DSB). These DSB are then corrected either by nonhomologous end joining

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Lipid nanoparticles loaded with ribonucleoprotein–oligonucleotide complexes synthesized using a microfluidic device exhibit robust genome editing and hepatitis B virus inhibition

Cited by 70 publications

References 53 publications

Oligonucleotide Therapies in the Treatment of Arthritis: A Narrative Review

Oligonucleotide Therapies in the Treatment of Arthritis: A Narrative Review

The Application of CRISPR/Cas Systems for Antiviral Therapy

The Delivery Challenge of Genome Editing in Human Epithelia

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