Abstract
In the last years the rapid development of Nucleic Acid Based Drugs (NABDs) to be used in gene therapy has had a great impact in the medical field, holding enormous promise, becoming “the latest generation medicine” with the first ever siRNA-lipid based formulation approved by the United States Food and Drug Administration (FDA) for human use, and currently on the market under the trade name Onpattro™. The growth of such powerful biologic therapeutics has gone hand in hand with the progress in delivery systems technology, which is absolutely required to improve their safety and effectiveness. Lipid carrier systems, particularly liposomes, have been proven to be the most suitable vehicles meeting NABDs requirements in the medical healthcare framework, limiting their toxicity, and ensuring their delivery and expression into the target tissues. In this review, after a description of the several kinds of liposomes structures and formulations used for in vitro or in vivo NABDs delivery, the broad range of siRNA-liposomes production techniques are discussed in the light of the latest technological progresses. Then, the current status of siRNA-lipid delivery systems in clinical trials is addressed, offering an updated overview on the clinical goals and the next challenges of this new class of therapeutics which will soon replace traditional drugs.
Highlights
Gene therapy is the treatment of diseases at molecular level by “switching genes on or off” through the use of Nucleic Acid Based Drugs (NABDs) which, including oligodeoxynucleotides, plasmid DNA, ribozymes, small interfering RNA (siRNA), miRNA and related chemically-synthesized molecules, are highly degradable therapeutics that need to be loaded into a vehicle in order to reach the patient target cell/tissue where the defective or mutated gene has to be corrected [1,2]
In the last years the rapid development of Nucleic Acid Based Drugs (NABDs) to be used in gene therapy has had a great impact in the medical field, holding enormous promise, becoming “the latest generation medicine” with the first ever siRNA-lipid based formulation approved by the United States Food and Drug Administration (FDA) for human use, and currently on the market under the trade name OnpattroTM
Gene therapy is the treatment of diseases at molecular level by “switching genes on or off” through the use of Nucleic Acid Based Drugs (NABDs) which, including oligodeoxynucleotides, plasmid DNA, ribozymes, siRNA, miRNA and related chemically-synthesized molecules, are highly degradable therapeutics that need to be loaded into a vehicle in order to reach the patient target cell/tissue where the defective or mutated gene has to be corrected [1,2]
Summary
Gene therapy is the treatment of diseases at molecular level by “switching genes on or off” through the use of Nucleic Acid Based Drugs (NABDs) which, including oligodeoxynucleotides, plasmid DNA, ribozymes, siRNA, miRNA and related chemically-synthesized molecules, are highly degradable therapeutics that need to be loaded into a vehicle in order to reach the patient target cell/tissue where the defective or mutated gene has to be corrected [1,2]. PPrroodduucceedd bbyy AAllnnyyllaamm PPhhaarrmmaacceeuuttiiccaallss CCoorrpporation and sold under the ttrrade nnaame OOnpattroTMTM,, AALLNN--TTTTRR0022 iiss aann ssiiRRNNAA ffoorrmmuullaattiioonn bbaasseedd uuppoonn SSttaabbllee NNuucclleeiicc AAcciidd LLiippiidd PPaarrttiiccllee ((SSNNAALLPP)) ttrraannssffeeccttiinnggtteecchhnnoollooggyy, ,aannddiissiinnddiiccaatteeddffoorrtthheettrreeaattmmeenntt ooff tthhee HHeerreeddiittaarryy TTrraannsstthhyyrreettiinn--mmeeddiiaatteedd AAmmyyllooiiddoossiiss ((hhAATTTTRR)),, a progressively iinnccaappaacciittaattiinnggaannddooftfetennfafatatal lggeneneteitcicdidsiosrodredre.rT. SScchheemmaattiizzaattiioonn ooff lliippiidd--bbaasseedd nnaannooppaarrttiicclleess tthhaatt aarree uusseedd ffoorr NNuucclleeiicc AAcciiddBBaasseeddDDrruuggss ((NNAABBDDss))ddeelilviveerryyaannddtthheeirirpprroodduucctitoionntetecchhnniqiquueess. TThheeiirr tthheerraappeeuuttiicc ppootteennttiiaall aass aa nneexxtt ggeenneerraattiioonn mmeeddiicciinnee hhaass bbeeeenn rreecceennttllyy rreevviieewweedd [[2222]],, ttooggeetthheerr wwiitthh tthhee mmaajjoorr ddeelliivveerryy ssttrraatteeggiieess ccuurrrreennttllyy aaddoopptteedd ffoorr tthheeiirr eeffffeeccttiivvee iinn vviittrroo aanndd iinn vviivvoo ddeelliivveerryy[[2233]]. AAfftteerr,,aaddiissccuussssiioonnaabboouuttlliippoossoommeessssttrruuccttuurreess,,tthheeffoorrmmuullaattiioonnssaannddpprroodduuccttiioonntteecchhnniiqquueess ssuuiittaabblelefofrorNANBADBsDdseldiveelirvyearrye arerpeorretepdo.rtIendt.heInlastht epalrats, tthpeamrte, dtihceal mapepdliiccaaltioanpspolifcsaitRioNnAs -olifpossiRomNeAscluiproresnotmlyeus ncudrerreinntvlyesutingdaetiroinnvinesctliignaictiaolntriinalcslianriecathl etrniatlrseaatreedthinendetrteaailt.ed in detail
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