Quick nuclear transportation of siRNA and in vivo hepatic ApoB gene silencing with galactose-bearing polymeric carrier

Tachibana, Yoichi; Munisso, Maria Chiara; Kamata, Wakako; Kitagawa, Masaru; Harada‐Shiba, Mariko; Yamaoka, Tetsuji

doi:10.1016/j.jbiotec.2014.01.029

Cited by 8 publications

(2 citation statements)

References 35 publications

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“…The incorporation of therapeutic molecules along with peptides shows the best therapeutic efficacy and less toxicity in nuclear targeting drug delivery systems . Over the last decades many peptides have been reported which show great potential in targeting the nucleus and penetrating the nuclear membrane. − …”

Section: Nucleus Targetingmentioning

confidence: 99%

Biomaterials and Bioengineering Approaches for Mitochondria and Nuclear Targeting Drug Delivery

Nurunnabi

Khatun

Badruddoza

et al. 2019

ACS Biomater. Sci. Eng.

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When a therapeutic molecule is administered into a biological system, it faces numerous barriers on its way to the target site. While a portion of the administered dose can overcome these barriers, localize to the targeted site, and provide a therapeutic effect, the vast majority either accumulates off-target sites or is excreted without pharmacological benefit. Current drug delivery research aims to address this problem by protecting therapeutic molecules from the biological milieu so that they can minimize off-target toxicity, while concomitantly overcoming the associated barriers to effectively reach the location of interest. In this review, we focus biomaterials and bioengineering approaches that have potential to overcome intracellular barriers. We discuss the various systems/materials developed so far to deliver drugs and genes and the major challenges that remain unmet. Finally, we provide outlook on the importance of subcellular targeting drug delivery and give an overview on potential approaches and materials.

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Section: Nucleus Targetingmentioning

confidence: 99%

Biomaterials and Bioengineering Approaches for Mitochondria and Nuclear Targeting Drug Delivery

Nurunnabi

Khatun

Badruddoza

et al. 2019

ACS Biomater. Sci. Eng.

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“…Although viral vectors provide an efficient delivery option, concerns persist regarding host immune response (Miele et al, 2012). Numerous non-viral strategies have been pursued including the use of peptides (Andaloussi et al, 2011), aptamers (Li et al, 2013), antibody-protamine chimeras (Song et al, 2005; Dou et al, 2012), cationic lipids (Morrissey et al, 2005; Sørensen and Sioud, 2010; Semple et al, 2010) and cationic polymers (Urban-Klein et al, 2005; Tachibana et al, 2014) as siRNA carriers. Cationic lipid-based stable nucleic acid particles (Barros and Gollob, 2012) and cyclodextrin nanoparticles (Davis et al, 2010) are currently in clinical trial as potential siRNA carriers.…”

Section: Introductionmentioning

confidence: 99%

Cationic lipid nanodisks as an siRNA delivery vehicle

Ghosh¹,

Ren

Simonsen³

et al. 2014

Biochem. Cell Biol.

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The term nanodisk (ND) describes reconstituted high-density lipoprotein particles that contain one or more exogenous bioactive agents. In the present study ND were assembled from apolipoprotein A-I, the zwitterionic glycerophospholipid, dimyristoylphosphatidylcholine (DMPC) and the synthetic cationic lipid, dimyristoyltrimethylaminopropane (DMTAP). ND formulated at a 70:30 weight ratio of DMPC to DMTAP were soluble in aqueous media. The particles generated were polydisperse, with diameters ranging from ~20 to <50 nm. In nucleic acid binding studies, agarose gel retardation assays revealed that a synthetic 23mer double-stranded oligonucleotide (dsOligo) bound to DMTAP containing ND but not to ND formulated with DMPC alone. Sucrose density gradient ultracentrifugation studies provided additional evidence for stable dsOligo binding to DMTAP-ND. Incubation of cultured hepatoma cells with DMTAP-ND complexed with a small interfering (si) RNA directed against glyceraldehyde 3-phosphate dehydrogenase showed 60 % knockdown efficiency. Thus, incorporation of synthetic cationic lipid (i.e. DMTAP) to ND confers an ability to bind siRNA and the resulting complexes possess target gene knockdown activity in a cultured cell model.

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Synthetic cationic glycopolymers for gene delivery

Ahmed

Narain

2016

Polymers and Nanomaterials for Gene Therapy

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Quick nuclear transportation of siRNA and in vivo hepatic ApoB gene silencing with galactose-bearing polymeric carrier

Cited by 8 publications

References 35 publications

Biomaterials and Bioengineering Approaches for Mitochondria and Nuclear Targeting Drug Delivery

Biomaterials and Bioengineering Approaches for Mitochondria and Nuclear Targeting Drug Delivery

Cationic lipid nanodisks as an siRNA delivery vehicle

Synthetic cationic glycopolymers for gene delivery

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