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Engineering liposomal nanoparticles for targeted gene therapy

Recent mechanistic studies have attempted to deepen our understanding of the process by which liposome-mediated delivery of genetic material occurs. Understanding the interactions between lipid nanoparticles and cells is still largely elusive. Liposome-mediated delivery of genetic material faces sys... Full description

Journal Title: Gene Therapy Aug 2017, Vol.24(8), pp.441-452
Main Author: Zylberberg, C
Other Authors: Gaskill, K , Pasley, S , Matosevic, S
Format: Electronic Article Electronic Article
Language: English
Subjects:
ID: ISSN: 09697128 ; E-ISSN: 14765462 ; DOI: 10.1038/gt.2017.41
Link: http://search.proquest.com/docview/1929446703/?pq-origsite=primo
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title: Engineering liposomal nanoparticles for targeted gene therapy
format: Article
creator:
  • Zylberberg, C
  • Gaskill, K
  • Pasley, S
  • Matosevic, S
subjects:
  • Gene Therapy
  • Engineering
  • Liposomes
  • Escape Systems
  • Degradation
  • Nanoparticles
  • Gene Therapy
  • Genetic Engineering
  • Nanoparticles
  • Lipids
  • Transfection
  • Liposomes
  • Chemical Bonds
  • Gene Therapy
  • Lysosomes
  • Nanoparticles
  • Deoxyribonucleic Acid–DNA
  • Deoxyribonucleic Acid–DNA
  • Deoxyribonucleic Acid–DNA
ispartof: Gene Therapy, Aug 2017, Vol.24(8), pp.441-452
description: Recent mechanistic studies have attempted to deepen our understanding of the process by which liposome-mediated delivery of genetic material occurs. Understanding the interactions between lipid nanoparticles and cells is still largely elusive. Liposome-mediated delivery of genetic material faces systemic obstacles alongside entry into the cell, endosomal escape, lysosomal degradation and nuclear uptake. Rational design approaches for targeted delivery have been developed to reduce off-target effects and enhance transfection. These strategies, which have included the modification of lipid nanoparticles with target-specific ligands to enhance intracellular uptake, have shown significant promise at the proof-of-concept stage. Control of physical and chemical specifications of liposome composition, which includes lipid-to-DNA charge, size, presence of ester bonds, chain length and nature of ligand complexation, is integral to the performance of targeted liposomes as genetic delivery agents. Clinical advances are expected to rely on such systems in the therapeutic application of liposome nanoparticle-based gene therapy. Here, we discuss the latest breakthroughs in the development of targeted liposome-based agents for the delivery of genetic material, paying particular attention to new ligand and cationic lipid design as well as recent in vivo advances.
language: eng
source:
identifier: ISSN: 09697128 ; E-ISSN: 14765462 ; DOI: 10.1038/gt.2017.41
fulltext: fulltext
issn:
  • 09697128
  • 0969-7128
  • 14765462
  • 1476-5462
url: Link


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titleEngineering liposomal nanoparticles for targeted gene therapy
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subjectGene Therapy ; Engineering ; Liposomes ; Escape Systems ; Degradation ; Nanoparticles ; Gene Therapy ; Genetic Engineering ; Nanoparticles ; Lipids ; Transfection ; Liposomes ; Chemical Bonds ; Gene Therapy ; Lysosomes ; Nanoparticles ; Deoxyribonucleic Acid–DNA ; Deoxyribonucleic Acid–DNA ; Deoxyribonucleic Acid–DNA
descriptionRecent mechanistic studies have attempted to deepen our understanding of the process by which liposome-mediated delivery of genetic material occurs. Understanding the interactions between lipid nanoparticles and cells is still largely elusive. Liposome-mediated delivery of genetic material faces systemic obstacles alongside entry into the cell, endosomal escape, lysosomal degradation and nuclear uptake. Rational design approaches for targeted delivery have been developed to reduce off-target effects and enhance transfection. These strategies, which have included the modification of lipid nanoparticles with target-specific ligands to enhance intracellular uptake, have shown significant promise at the proof-of-concept stage. Control of physical and chemical specifications of liposome composition, which includes lipid-to-DNA charge, size, presence of ester bonds, chain length and nature of ligand complexation, is integral to the performance of targeted liposomes as genetic delivery agents. Clinical advances are expected to rely on such systems in the therapeutic application of liposome nanoparticle-based gene therapy. Here, we discuss the latest breakthroughs in the development of targeted liposome-based agents for the delivery of genetic material, paying particular attention to new ligand and cationic lipid design as well as recent in vivo advances.
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abstractRecent mechanistic studies have attempted to deepen our understanding of the process by which liposome-mediated delivery of genetic material occurs. Understanding the interactions between lipid nanoparticles and cells is still largely elusive. Liposome-mediated delivery of genetic material faces systemic obstacles alongside entry into the cell, endosomal escape, lysosomal degradation and nuclear uptake. Rational design approaches for targeted delivery have been developed to reduce off-target effects and enhance transfection. These strategies, which have included the modification of lipid nanoparticles with target-specific ligands to enhance intracellular uptake, have shown significant promise at the proof-of-concept stage. Control of physical and chemical specifications of liposome composition, which includes lipid-to-DNA charge, size, presence of ester bonds, chain length and nature of ligand complexation, is integral to the performance of targeted liposomes as genetic delivery agents. Clinical advances are expected to rely on such systems in the therapeutic application of liposome nanoparticle-based gene therapy. Here, we discuss the latest breakthroughs in the development of targeted liposome-based agents for the delivery of genetic material, paying particular attention to new ligand and cationic lipid design as well as recent in vivo advances.
copHoundmills
pubNature Publishing Group
doi10.1038/gt.2017.41
urlhttp://search.proquest.com/docview/1929446703/
date2017-08-01