Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides

Keith Henry Moss, Petya Popova, Sine Reker Hadrup, Kira Astakhova*, Maria Taskova

*Corresponding author for this work

Research output: Contribution to journalJournal articleResearchpeer-review

Abstract

Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNPs). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and screening LNP candidates for effective translation into drugs of human metabolic diseases and cancer.
Original languageEnglish
JournalMolecular Pharmaceutics
Volume16
Issue number6
Pages (from-to)2265-2277
Number of pages13
ISSN1543-8384
DOIs
Publication statusPublished - 2019

Keywords

  • Gene therapy
  • RNA
  • Delivery
  • Lipid nanoparticle (LNP)
  • LNP fabrication
  • LNP screening

Cite this

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title = "Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides",
abstract = "Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNPs). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and screening LNP candidates for effective translation into drugs of human metabolic diseases and cancer.",
keywords = "Gene therapy, RNA, Delivery, Lipid nanoparticle (LNP), LNP fabrication, LNP screening",
author = "Moss, {Keith Henry} and Petya Popova and Hadrup, {Sine Reker} and Kira Astakhova and Maria Taskova",
year = "2019",
doi = "10.1021/acs.molpharmaceut.8b01290",
language = "English",
volume = "16",
pages = "2265--2277",
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publisher = "American Chemical Society",
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Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides. / Moss, Keith Henry; Popova, Petya; Hadrup, Sine Reker; Astakhova, Kira; Taskova, Maria.

In: Molecular Pharmaceutics, Vol. 16, No. 6, 2019, p. 2265-2277.

Research output: Contribution to journalJournal articleResearchpeer-review

TY - JOUR

T1 - Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides

AU - Moss, Keith Henry

AU - Popova, Petya

AU - Hadrup, Sine Reker

AU - Astakhova, Kira

AU - Taskova, Maria

PY - 2019

Y1 - 2019

N2 - Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNPs). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and screening LNP candidates for effective translation into drugs of human metabolic diseases and cancer.

AB - Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNPs). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and screening LNP candidates for effective translation into drugs of human metabolic diseases and cancer.

KW - Gene therapy

KW - RNA

KW - Delivery

KW - Lipid nanoparticle (LNP)

KW - LNP fabrication

KW - LNP screening

U2 - 10.1021/acs.molpharmaceut.8b01290

DO - 10.1021/acs.molpharmaceut.8b01290

M3 - Journal article

C2 - 31063396

VL - 16

SP - 2265

EP - 2277

JO - Molecular Pharmaceutics

JF - Molecular Pharmaceutics

SN - 1543-8384

IS - 6

ER -