Lipid nanoparticles for targeted siRNA delivery – going from bench to bedside

References

• VinoresSAPegaptanib in the treatment of wet, age-related macular degenerationInt J Nanomedicine20061326326817717967
   PubMed Web of Science ®Google Scholar
• U.S. Food and Drug AdministrationFDA Approves New Drug Treatment for Age-Related Macular Degeneration [press release]2004 [December 20]. Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2004/ucm108385.htmAccessed December 1, 2015
   Google Scholar
• TseMTRegulatory watch: Antisense approval provides boost to the fieldNature Rev Drug Disc201312179
   Google Scholar
• U.S. Food and Drug AdministrationFDA approves new orphan drug Kynamro to treat inherited cholesterol disorder [press release]2013 [January 29]. Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm337195.htmAccessed December 1, 2015
   Google Scholar
• ZuckermanJEDavisMEClinical experiences with systemically administered siRNA-based therapeutics in cancerNat Rev Drug Discov2015141284385626567702
   PubMed Web of Science ®Google Scholar
• BobbinMLRossiJJRNA interference (RNAi)-based therapeutics: delivering on the promise?Annu Rev Pharmacol Toxicol20165610312226738473
   PubMed Web of Science ®Google Scholar
• LamJKChowMYZhangYLeungSWsiRNA versus miRNA as therapeutics for gene silencingMol Ther Nucleic Acids20154e25226372022
   PubMedGoogle Scholar
• ChakrabortyCWenZHAgoramoorthyGLinCSTherapeutic microRNA delivery strategies with special emphasis on cancer therapy and tumorigenesis: current trends and future challengesCurr Drug Metab201617546947726813887
   PubMed Web of Science ®Google Scholar
• WittrupALiebermanJKnocking down disease: a progress report on siRNA therapeuticsNat Rev Genet201516954355226281785
   PubMed Web of Science ®Google Scholar
• SahinUKarikóKTüreciÖmRNA-based therapeutics – developing a new class of drugsNat Rev Drug Discov2014131075978025233993
   PubMed Web of Science ®Google Scholar
• KumarVMahatoRIDelivery and targeting of miRNAs for treating liver fibrosisPharm Res201532234136125186440
   PubMedGoogle Scholar
• KanastyRDorkinJRVegasAAndersonDDelivery materials for siRNA therapeuticsNat Mater2013121196797724150415
   PubMed Web of Science ®Google Scholar
• YinHKanastyRLEltoukhyAAVegasAJDorkinJRAndersonDGNon-viral vectors for gene-based therapyNat Rev Genet201415854155525022906
   PubMed Web of Science ®Google Scholar
• SarettSMNelsonCEDuvallCLTechnologies for controlled, local delivery of siRNAJ Control Release20152189411326476177
   PubMed Web of Science ®Google Scholar
• FujitaYKuwanoKOchiyaTDevelopment of small RNA delivery systems for lung cancer therapyInt J Mol Sci20151635254527025756380
   PubMed Web of Science ®Google Scholar
• SinghYTomarSKhanSBridging small interfering RNA with giant therapeutic outcomes using nanometric liposomesJ Control Release2015220Pt A36838726528900
   PubMedGoogle Scholar
• LeungAKTamYYCullisPRLipid nanoparticles for short interfering RNA deliveryAdv Genet2014887111025409604
   PubMed Web of Science ®Google Scholar
• HopeMJEnhancing siRNA delivery by employing lipid nanoparticlesTher Deliv20145666367325090280
   PubMedGoogle Scholar
• WanCAllenTMCullisPRLipid nanoparticle delivery systems for siRNA-based therapeuticsDrug Deliv Transl Res201441748325786618
   PubMed Web of Science ®Google Scholar
• MaierMAJayaramanMMatsudaSBiodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeuticsMol Ther20132181570157823799535
   PubMed Web of Science ®Google Scholar
• LoveKTMahonKPLevinsCGLipid-like materials for low-dose, in vivo gene silencingProc Natl Acad Sci U S A201010751864186920080679
   PubMed Web of Science ®Google Scholar
• NovobrantsevaTIBorodovskyAWongJSystemic RNAi-mediated gene silencing in nonhuman primate and rodent myeloid cellsMol Ther Nucleic Acids20121e423344621
   PubMedGoogle Scholar
• ChengXLeeRJThe role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide deliveryAdv Drug Deliv Rev201699Pt A12913726900977
   PubMed Web of Science ®Google Scholar
• KumarVQinJJiangYShielding of lipid nanoparticles for siRNA delivery: impact on physicochemical properties, cytokine induction, and efficacyMol Ther Nucleic Acids20143e21025405467
   PubMedGoogle Scholar
• XiaYTianJChenXEffect of surface properties on liposomal siRNA deliveryBiomaterials201679566826695117
   PubMed Web of Science ®Google Scholar
• DabkowskaAPBarlowDJHughesAVCampbellRAQuinnPJLawrenceMJThe effect of neutral helper lipids on the structure of cationic lipid monolayersJ R Soc Interface201296854856121831895
   PubMedGoogle Scholar
• TenchovBGMacDonaldRCSiegelDPCubic phases in phosphatidylcholine-cholesterol mixtures: cholesterol as membrane “fusogen”Biophys J20069172508251616829556
   PubMedGoogle Scholar
• ErnstingMJMurakamiMRoyALiSDFactors controlling the pharmacokinetics, biodistribution and intratumoral penetration of nanoparticlesJ Control Release2013172378279424075927
   PubMed Web of Science ®Google Scholar
• GilleronJQuerbesWZeigererAImage-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escapeNat Biotechnol201331763864623792630
   PubMed Web of Science ®Google Scholar
• SahayGQuerbesWAlabiCEfficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recyclingNat Biotechnol201331765365823792629
   PubMed Web of Science ®Google Scholar
• GilleronJParamasivamPZeigererAIdentification of siRNA delivery enhancers by a chemical library screenNucleic Acids Res201543167984800126220182
   PubMedGoogle Scholar
• OsbornMFAltermanJFNikanMGuanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAsNucleic Acids Res201543188664867226400165
   PubMedGoogle Scholar
• HouKKPanHSchlesingerPHWicklineSAA role for peptides in overcoming endosomal entrapment in siRNA delivery – a focus on melittinBiotechnol Adv2015336 Pt 193194026025036
   PubMed Web of Science ®Google Scholar
• HouKKPanHRatnerLSchlesingerPHWicklineSAMechanisms of nanoparticle-mediated siRNA transfection by melittin-derived peptidesACS Nano20137108605861524053333
   PubMed Web of Science ®Google Scholar
• WooddellCIRozemaDBHossbachMHepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infectionMol Ther201321597398523439496
   PubMed Web of Science ®Google Scholar
• GolovinYIGribanovskySLGolovinDYTowards nanomedicines of the future: Remote magneto-mechanical actuation of nanomedicines by alternating magnetic fieldsJ Control Release2015219436026407671
   PubMed Web of Science ®Google Scholar
• AkincAQuerbesWDeSTargeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanismsMol Ther20101871357136420461061
   PubMed Web of Science ®Google Scholar
• CohenZRRamishettiSPeshes-YalozNLocalized RNAi therapeutics of chemoresistant grade IV glioma using hyaluronan-grafted lipid-based nanoparticlesACS Nano2015921581159125558928
   PubMed Web of Science ®Google Scholar
• McCaskillJSinghaniaRBurgessMEfficient biodistribution and gene silencing in the lung epithelium via intravenous liposomal delivery of siRNAMol Ther Nucleic Acids20132e9623736774
   PubMedGoogle Scholar
• HajduPChimoteAAThompsonTHKooYYunYConfortiLFunctionalized liposomes loaded with siRNAs targeting ion channels in effector memory T cells as a potential therapy for autoimmunityBiomaterials20133438102491025724075407
   PubMedGoogle Scholar
• RamishettiSKedmiRGoldsmithMSystemic gene silencing in primary T lymphocytes using targeted lipid nanoparticlesACS Nano2015976706671626042619
   PubMedGoogle Scholar
• WeinsteinSTokerIAEmmanuelRHarnessing RNAi-based nanomedicines for therapeutic gene silencing in B-cell malignanciesProc Natl Acad Sci U S A20161131E16E2226699502
   PubMedGoogle Scholar
• KuSHJoSDLeeYKKimKKimSHChemical and structural modifications of RNAi therapeuticsAdv Drug Deliv Rev Epub2015115
   Google Scholar
• ChenSTamYYLinPJLeungAKTamYKCullisPRDevelopment of lipid nanoparticle formulations of siRNA for hepatocyte gene silencing following subcutaneous administrationJ Control Release201419610611225285610
   PubMed Web of Science ®Google Scholar
• Landesman-MiloDPeerDTransforming nanomedicines from lab scale production to novel clinical modalityBioconjug Chem Epub2016120
   Google Scholar
• SempleSCAkincAChenJRational design of cationic lipids for siRNA deliveryNat Biotechnol201028217217620081866
   PubMed Web of Science ®Google Scholar
• TousignantJDGatesALIngramLAComprehensive analysis of the acute toxicities induced by systemic administration of cationic lipid:plasmid DNA complexes in miceHum Gene Ther200011182493251311119421
   PubMed Web of Science ®Google Scholar
• ZhangJSLiuFHuangLImplications of pharmacokinetic behavior of lipoplex for its inflammatory toxicityAdv Drug Deliv Rev200557568969815757755
   PubMed Web of Science ®Google Scholar
• XueHYLiuSWongHLNanotoxicity: a key obstacle to clinical translation of siRNA-based nanomedicineNanomedicine (Lond)20149229531224552562
   PubMed Web of Science ®Google Scholar
• KimHPHanSWSongSHTestican-1-mediated epithelial-mesenchymal transition signaling confers acquired resistance to lapatinib in HER2-positive gastric cancerOncogene201433253334334123873022
   PubMed Web of Science ®Google Scholar
• ZhouDZhangYILiangDEffect of combination therapy of siRNA targeting growth hormone receptor and 5-fluorouracil in hepatic metastasis of colon cancerOncol Lett20151063505350926788158
   PubMedGoogle Scholar
• GeisbertTWLeeACRobbinsMPostexposure protection of non-human primates against a lethal Ebola virus challenge with RNA interference: a proof-of-concept studyLancet201037597291896190520511019
   PubMed Web of Science ®Google Scholar
• YuenMFChanHLGivenBDHamiltonJSchluepTDavidLPhase II, dose ranging study of ARC-520, a siRNA-based therapeutic, in patients with chronic hepatitis B virus infectionHepatology2015621385A
   Web of Science ®Google Scholar
• YuenMFChanHLGivenBDARC-520 produces deep and durable knockdown of viral antigens and DNA in a phase II study in patients with chronic hepatitis BHepatology2015621385A
   Web of Science ®Google Scholar
• GishRGYuenMFChanHLSynthetic RNAi triggers and their use in chronic hepatitis B therapies with curative intentAntiviral Res20151219710826129970
   PubMed Web of Science ®Google Scholar
• BegMSBrennerASachdevJSafety, tolerability, and clinical activity of MRX34, the first-in-class liposomal miR-34 mimic, in patients with advanced solid tumorsMol Cancer Ther201514C43
   Google Scholar
• LiuXTargeting polo-like kinases: a promising therapeutic approach for cancer treatmentTransl Oncol20158318519526055176
   PubMed Web of Science ®Google Scholar
• RamanathanRKHamburgSIBoradMJA phase I dose escalation study of TKM-080301, a RNAi therapeutic directed against PLK1, in patients with advanced solid tumorsCancer Res2013738 SupplLB-289
   Google Scholar
• AbramsMGaneshSYingBEnCore-LNP mediated tumor delivery of MYC and CTNNB1 dicer substrate RNAs (DsiRNAs)Mol Cancer Res20151310 SupplB20
   Google Scholar
• GaneshSYingBKoserMSystemic delivery of CTNNB1 dicer-substrate siRNAs (DsiRNAs) leads to efficient oncogene silencing in diverse tumor types of extra hepatic originCancer Res20157515 Suppl3533
   Google Scholar
• LandenCNJrChavez-ReyesABucanaCTherapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA deliveryCancer Res200565156910691816061675
   PubMed Web of Science ®Google Scholar
• ShenHRodriguez-AguayoCXuREnhancing chemotherapy response with sustained EphA2 silencing using multistage vector deliveryClin Cancer Res20131971806181523386691
   PubMedGoogle Scholar
• OzcanGOzpolatBColemanRLSoodAKLopez-BeresteinGPreclinical and clinical development of siRNA-based therapeuticsAdv Drug Deliv Rev20158710811925666164
   PubMed Web of Science ®Google Scholar
• FitzgeraldKFrank-KamenetskyMShulga-MorskayaSEffect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, Phase 1 trialLancet20143839911606824094767
   PubMed Web of Science ®Google Scholar
• HortonJDCohenJCHobbsHHMolecular biology of PCSK9: its role in LDL metabolismTrends Biochem Sci2007322717717215125
   PubMed Web of Science ®Google Scholar
• PoirierSMayerGPouponVDissection of the endogenous cellular pathways of PCSK9-induced low density lipoprotein receptor degradation: evidence for an intracellular routeJ Biol Chem200928442288562886419635789
   PubMed Web of Science ®Google Scholar
• ZhangXLZhuQQZhuLSafety and efficacy of anti-PCSK9 antibodies: a meta-analysis of 25 randomized, controlled trialsBMC Med20151312326099511
   PubMed Web of Science ®Google Scholar
• JørgensenABFrikke-SchmidtRNordestgaardBGTybjærg-Hansenaloss-of-function mutations in APOC3 and risk of ischemic vascular diseaseN Engl J Med20143711324124941082
   PubMed Web of Science ®Google Scholar
• GrahamMJLeeRGBellTA3rdAntisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humansCirc Res2013112111479149023542898
   PubMed Web of Science ®Google Scholar
• WangXWangDShanZClinical and genetic analysis of a family diagnosed with familial hypobetalipoproteinemia in which the proband was diagnosed with diabetes mellitusAtherosclerosis2015239255255625733326
   PubMedGoogle Scholar
• TikkaASoronenJLaurilaPPMetsoJEhnholmCJauhiainenMSilencing of ANGPTL 3 (angiopoietin-like protein 3) in human hepatocytes results in decreased expression of gluconeogenic genes and reduced triacylglycerol-rich VLDL secretion upon insulin stimulationBiosci Rep2014346e0016025495645
   PubMedGoogle Scholar
• CoelhoTAdamsDSilvaASafety and efficacy of RNAi therapy for transthyretin amyloidosisN Engl J Med2013369981982923984729
   PubMed Web of Science ®Google Scholar
• ButlerJSChanACostelhaSPreclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosisAmyloid201623211026646718
   PubMedGoogle Scholar
• HaaseNFosterDBercherJRNAi therapeutics targeting human angiotensinogen (hAGT) ameliorate preeclamptic sequelae in an established transgenic rodent model for preeclampsiaHypertension201464A666
   Google Scholar