Technologies for Investigating the Physiological Barriers to Efficient Lipid Nanoparticle-siRNA Delivery

被引：21
作者：
Shi, Bin [1 ]
Abrams, Marc [1 ]
机构：
[1] Merck & Co Inc, Merck Res Labs, Dept RNA Therapeut, West Point, PA USA
来源：
JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY | 2013年 / 61卷 / 06期
关键词：
lipid nanoparticle; LNP; siRNA; biodistribution; QWBA; intravital imaging; stem-loop PCR; RISC; endosomal escape; delivery; immunofluorescence staining; SMALL-INTERFERING RNA; B-VIRUS REPLICATION; IN-VIVO DELIVERY; PROOF-OF-CONCEPT; LOOP RT-PCR; GENE DELIVERY; NONHUMAN-PRIMATES; TARGETED DELIVERY; SYSTEMIC DELIVERY; ANTISENSE OLIGONUCLEOTIDES;
D O I：
10.1369/0022155413484152
中图分类号：
Q2 [细胞生物学];
学科分类号：
071009 ; 090102 ;
摘要：
Small interfering RNA (siRNA) therapeutics have advanced from bench to clinical trials in recent years, along with new tools developed to enable detection of siRNA delivered at the organ, cell, and subcellular levels. Preclinical models of siRNA delivery have benefitted from methodologies such as stem-loop quantitative polymerase chain reaction, histological in situ immunofluorescent staining, endosomal escape assay, and RNA-induced silencing complex loading assay. These technologies have accelerated the detection and optimization of siRNA platforms to overcome the challenges associated with delivering therapeutic oligonucleotides to the cytosol of specific target cells. This review focuses on the methodologies and their application in the biodistribution of siRNA delivered by lipid nanoparticles.
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收藏
页码：407 / 420
页数：14
相关论文
共 139 条
  • [1] Evaluation of Efficacy, Biodistribution, and Inflammation for a Potent siRNA Nanoparticle: Effect of Dexamethasone Co-treatment
    Abrams, Marc T.
    Koser, Martin L.
    Seitzer, Jessica
    Williams, Stephanie C.
    DiPietro, Martha A.
    Wang, Weimin
    Shaw, Andrew W.
    Mao, Xianzhi
    Jadhav, Vasant
    Davide, Joseph P.
    Burke, Paul A.
    Sachs, Alan B.
    Stirdivant, Steven M.
    Sepp-Lorenzino, Laura
    [J]. MOLECULAR THERAPY, 2010, 18 (01) : 171 - 180
  • [2] Nanoparticles for ex vivo siRNA delivery to dendritic cells for cancer vaccines: Programmed endosomal escape and dissociation
    Akita, Hidetaka
    Kogure, Kentaro
    Moriguchi, Rumiko
    Nakamura, Yoshio
    Higashi, Tomoko
    Nakamura, Takashi
    Serada, Satoshi
    Fujimoto, Minoru
    Naka, Tetsuji
    Futaki, Shiroh
    Harashima, Hideyoshi
    [J]. JOURNAL OF CONTROLLED RELEASE, 2010, 143 (03) : 311 - 317
  • [3] Microcalorimetric Method to Assess Phagocytosis: Macrophage-Nanoparticle Interactions
    Al-Hallak, M. H. D. Kamal
    Sarfraz, Muhammad Khan
    Azarmi, Shirzad
    Kohan, M. H. Gilzad
    Roa, Wilson H.
    Loebenberg, Raimar
    [J]. AAPS JOURNAL, 2011, 13 (01): : 20 - 29
  • [4] Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation
    Aouadi, Myriam
    Tesz, Gregory J.
    Nicoloro, Sarah M.
    Wang, Mengxi
    Chouinard, My
    Soto, Ernesto
    Ostroff, Gary R.
    Czech, Michael P.
    [J]. NATURE, 2009, 458 (7242) : 1180 - U116
  • [5] AROETI B, 1991, J BIOL CHEM, V266, P15845
  • [6] Dicetyl Phosphate-Tetraethylenepentamine-Based Liposomes for Systemic siRNA Delivery
    Asai, Tomohiro
    Matsushita, Saori
    Kenjo, Eriya
    Tsuzuku, Takuma
    Yonenaga, Norihito
    Koide, Hiroyuki
    Hatanaka, Kentaro
    Dewa, Takehisa
    Nango, Mamoru
    Maeda, Noriyuki
    Kikuchi, Hiroshi
    Oku, Naoto
    [J]. BIOCONJUGATE CHEMISTRY, 2011, 22 (03) : 429 - 435
  • [7] Impact of tumor-specific targeting on the biodistribution and efficacy of siRNA nanoparticles measured by multimodality in vivo imaging
    Bartlett, Derek W.
    Su, Helen
    Hildebrandt, Isabel J.
    Weber, Wolfgang A.
    Davis, Mark E.
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2007, 104 (39) : 15549 - 15554
  • [8] Effective siRNA delivery and target mRNA degradation using an amphipathic peptide to facilitate pH-dependent endosomal escape
    Bartz, Rene
    Fan, Haihong
    Zhang, Jingtao
    Innocent, Nathalie
    Cherrin, Craig
    Beck, Stephen C.
    Pei, Yi
    Momose, Aaron
    Jadhav, Vasant
    Tellers, David M.
    Meng, Fanyu
    Crocker, Louis S.
    Sepp-Lorenzino, Laura
    Barnett, Stanley F.
    [J]. BIOCHEMICAL JOURNAL, 2011, 435 : 475 - 487
  • [9] Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells
    Basha, Genc
    Novobrantseva, Tatiana I.
    Rosin, Nicole
    Tam, Yuen Yi C.
    Hafez, Ismail M.
    Wong, Matthew K.
    Sugo, Tsukasa
    Ruda, Vera M.
    Qin, June
    Klebanov, Boris
    Ciufolini, Marco
    Akinc, Akin
    Tam, Ying K.
    Hope, Michael J.
    Cullis, Pieter R.
    [J]. MOLECULAR THERAPY, 2011, 19 (12) : 2186 - 2200
  • [10] Translational siRNA Therapeutics Using Liposomal Carriers: Prospects & Challenges
    Bhavsar, Dhiraj
    Subramanian, Krishnakumar
    Sethuraman, Swaminathan
    Krishnan, Uma Maheswari
    [J]. CURRENT GENE THERAPY, 2012, 12 (04) : 315 - 332
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