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Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

Genetic drugs based on RNA or DNA have remarkable therapeutic potential as virtually any disease can be treated by silencing a pathological gene, expressing a beneficial protein, or by editing defective genes. However, therapies based on nucleic acid polymers require sophisticated delivery systems t... Full description

Journal Title: Nucleic acid therapeutics 2018-06-01, Vol.28 (3), p.146-157
Main Author: Kulkarni, Jayesh A.
Other Authors: Cullis, Pieter R. , van der Meel, Roy
Format: Electronic Article Electronic Article
Language: English
Subjects:
DNA
RNA
Quelle: Alma/SFX Local Collection
Publisher: United States: Mary Ann Liebert, Inc
ID: ISSN: 2159-3337
Link: https://www.ncbi.nlm.nih.gov/pubmed/29683383
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title: Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility
format: Article
creator:
  • Kulkarni, Jayesh A.
  • Cullis, Pieter R.
  • van der Meel, Roy
subjects:
  • Amyloid Neuropathies, Familial - genetics
  • Amyloid Neuropathies, Familial - metabolism
  • Amyloid Neuropathies, Familial - pathology
  • Amyloid Neuropathies, Familial - therapy
  • Amyloidosis
  • Animals
  • Bioavailability
  • Biochemistry
  • Cations
  • Cell division
  • Clinical Trials as Topic
  • CRISPR
  • Deoxyribonucleic acid
  • DNA
  • Drug delivery
  • Drug delivery systems
  • Drug development
  • Drug Discovery
  • Drug dosages
  • Drugs
  • FDA approval
  • Gene therapy
  • Gene Transfer Techniques
  • Genes
  • genetic drugs
  • Genetic Therapy - methods
  • Genetics
  • Genomes
  • Humans
  • Hypercholesterolemia - genetics
  • Hypercholesterolemia - metabolism
  • Hypercholesterolemia - pathology
  • Hypercholesterolemia - therapy
  • Immune response
  • Immunosuppressive agents
  • ionizable cationic lipid
  • lipid nanoparticle
  • Lipids
  • Lipids - chemistry
  • Liver
  • Liver - metabolism
  • Liver - pathology
  • Liver - virology
  • Macromolecules
  • Melanoma - genetics
  • Melanoma - metabolism
  • Melanoma - pathology
  • Melanoma - therapy
  • Molecular Biology
  • Molecular Medicine
  • mRNA
  • Nanoparticles
  • Nanoparticles - administration & dosage
  • Nanoparticles - chemistry
  • Nanoparticles - metabolism
  • nucleic acid
  • Payloads
  • Polymers
  • Proteins
  • Regulatory agencies
  • Regulatory approval
  • Reviews
  • Ribonucleic acid
  • RNA
  • RNA, Small Interfering - genetics
  • RNA, Small Interfering - metabolism
  • RNA, Small Interfering - pharmacokinetics
  • RNA, Small Interfering - therapeutic use
  • siRNA
  • Skin Neoplasms - genetics
  • Skin Neoplasms - metabolism
  • Skin Neoplasms - pathology
  • Skin Neoplasms - therapy
  • Transthyretin
  • Virus Diseases - genetics
  • Virus Diseases - pathology
  • Virus Diseases - therapy
  • Virus Diseases - virology
ispartof: Nucleic acid therapeutics, 2018-06-01, Vol.28 (3), p.146-157
description: Genetic drugs based on RNA or DNA have remarkable therapeutic potential as virtually any disease can be treated by silencing a pathological gene, expressing a beneficial protein, or by editing defective genes. However, therapies based on nucleic acid polymers require sophisticated delivery systems to deliver these macromolecules to the interior of target cells. In this study, we review progress in developing nonviral lipid nanoparticle (LNP) delivery systems that have attractive properties, including ease of manufacture, reduced immune responses, multidosing capabilities, larger payloads, and flexibility of design. LNP systems represent the most advanced delivery systems for genetic drugs as it is expected that an LNP-short interfering RNA (siRNA) formulation will receive clinical approval from the Food and Drug Administration (FDA) in 2018 for treatment of the hereditary condition transthyretin-mediated amyloidosis, a fatal condition for which there is currently no treatment. This achievement is largely due to the development of optimized ionizable cationic lipids, arguably the most important factor in the clinical success of LNP-siRNA. In addition, we highlight potential LNP applications, including targeting tissues beyond the liver and therapeutic approaches based on messenger RNA or Clustered Regularly Interspaced Short Palindromic Repeats/Cas.
language: eng
source: Alma/SFX Local Collection
identifier: ISSN: 2159-3337
fulltext: fulltext
issn:
  • 2159-3337
  • 2159-3345
  • 1557-8526
url: Link


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descriptionGenetic drugs based on RNA or DNA have remarkable therapeutic potential as virtually any disease can be treated by silencing a pathological gene, expressing a beneficial protein, or by editing defective genes. However, therapies based on nucleic acid polymers require sophisticated delivery systems to deliver these macromolecules to the interior of target cells. In this study, we review progress in developing nonviral lipid nanoparticle (LNP) delivery systems that have attractive properties, including ease of manufacture, reduced immune responses, multidosing capabilities, larger payloads, and flexibility of design. LNP systems represent the most advanced delivery systems for genetic drugs as it is expected that an LNP-short interfering RNA (siRNA) formulation will receive clinical approval from the Food and Drug Administration (FDA) in 2018 for treatment of the hereditary condition transthyretin-mediated amyloidosis, a fatal condition for which there is currently no treatment. This achievement is largely due to the development of optimized ionizable cationic lipids, arguably the most important factor in the clinical success of LNP-siRNA. In addition, we highlight potential LNP applications, including targeting tissues beyond the liver and therapeutic approaches based on messenger RNA or Clustered Regularly Interspaced Short Palindromic Repeats/Cas.
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subjectAmyloid Neuropathies, Familial - genetics ; Amyloid Neuropathies, Familial - metabolism ; Amyloid Neuropathies, Familial - pathology ; Amyloid Neuropathies, Familial - therapy ; Amyloidosis ; Animals ; Bioavailability ; Biochemistry ; Cations ; Cell division ; Clinical Trials as Topic ; CRISPR ; Deoxyribonucleic acid ; DNA ; Drug delivery ; Drug delivery systems ; Drug development ; Drug Discovery ; Drug dosages ; Drugs ; FDA approval ; Gene therapy ; Gene Transfer Techniques ; Genes ; genetic drugs ; Genetic Therapy - methods ; Genetics ; Genomes ; Humans ; Hypercholesterolemia - genetics ; Hypercholesterolemia - metabolism ; Hypercholesterolemia - pathology ; Hypercholesterolemia - therapy ; Immune response ; Immunosuppressive agents ; ionizable cationic lipid ; lipid nanoparticle ; Lipids ; Lipids - chemistry ; Liver ; Liver - metabolism ; Liver - pathology ; Liver - virology ; Macromolecules ; Melanoma - genetics ; Melanoma - metabolism ; Melanoma - pathology ; Melanoma - therapy ; Molecular Biology ; Molecular Medicine ; mRNA ; Nanoparticles ; Nanoparticles - administration & dosage ; Nanoparticles - chemistry ; Nanoparticles - metabolism ; nucleic acid ; Payloads ; Polymers ; Proteins ; Regulatory agencies ; Regulatory approval ; Reviews ; Ribonucleic acid ; RNA ; RNA, Small Interfering - genetics ; RNA, Small Interfering - metabolism ; RNA, Small Interfering - pharmacokinetics ; RNA, Small Interfering - therapeutic use ; siRNA ; Skin Neoplasms - genetics ; Skin Neoplasms - metabolism ; Skin Neoplasms - pathology ; Skin Neoplasms - therapy ; Transthyretin ; Virus Diseases - genetics ; Virus Diseases - pathology ; Virus Diseases - therapy ; Virus Diseases - virology
ispartofNucleic acid therapeutics, 2018-06-01, Vol.28 (3), p.146-157
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descriptionGenetic drugs based on RNA or DNA have remarkable therapeutic potential as virtually any disease can be treated by silencing a pathological gene, expressing a beneficial protein, or by editing defective genes. However, therapies based on nucleic acid polymers require sophisticated delivery systems to deliver these macromolecules to the interior of target cells. In this study, we review progress in developing nonviral lipid nanoparticle (LNP) delivery systems that have attractive properties, including ease of manufacture, reduced immune responses, multidosing capabilities, larger payloads, and flexibility of design. LNP systems represent the most advanced delivery systems for genetic drugs as it is expected that an LNP-short interfering RNA (siRNA) formulation will receive clinical approval from the Food and Drug Administration (FDA) in 2018 for treatment of the hereditary condition transthyretin-mediated amyloidosis, a fatal condition for which there is currently no treatment. This achievement is largely due to the development of optimized ionizable cationic lipids, arguably the most important factor in the clinical success of LNP-siRNA. In addition, we highlight potential LNP applications, including targeting tissues beyond the liver and therapeutic approaches based on messenger RNA or Clustered Regularly Interspaced Short Palindromic Repeats/Cas.
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1Amyloid Neuropathies, Familial - metabolism
2Amyloid Neuropathies, Familial - pathology
3Amyloid Neuropathies, Familial - therapy
4Amyloidosis
5Animals
6Bioavailability
7Biochemistry
8Cations
9Cell division
10Clinical Trials as Topic
11CRISPR
12Deoxyribonucleic acid
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15Drug delivery systems
16Drug development
17Drug Discovery
18Drug dosages
19Drugs
20FDA approval
21Gene therapy
22Gene Transfer Techniques
23Genes
24genetic drugs
25Genetic Therapy - methods
26Genetics
27Genomes
28Humans
29Hypercholesterolemia - genetics
30Hypercholesterolemia - metabolism
31Hypercholesterolemia - pathology
32Hypercholesterolemia - therapy
33Immune response
34Immunosuppressive agents
35ionizable cationic lipid
36lipid nanoparticle
37Lipids
38Lipids - chemistry
39Liver
40Liver - metabolism
41Liver - pathology
42Liver - virology
43Macromolecules
44Melanoma - genetics
45Melanoma - metabolism
46Melanoma - pathology
47Melanoma - therapy
48Molecular Biology
49Molecular Medicine
50mRNA
51Nanoparticles
52Nanoparticles - administration & dosage
53Nanoparticles - chemistry
54Nanoparticles - metabolism
55nucleic acid
56Payloads
57Polymers
58Proteins
59Regulatory agencies
60Regulatory approval
61Reviews
62Ribonucleic acid
63RNA
64RNA, Small Interfering - genetics
65RNA, Small Interfering - metabolism
66RNA, Small Interfering - pharmacokinetics
67RNA, Small Interfering - therapeutic use
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75Virus Diseases - pathology
76Virus Diseases - therapy
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21Gene therapy
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abstractGenetic drugs based on RNA or DNA have remarkable therapeutic potential as virtually any disease can be treated by silencing a pathological gene, expressing a beneficial protein, or by editing defective genes. However, therapies based on nucleic acid polymers require sophisticated delivery systems to deliver these macromolecules to the interior of target cells. In this study, we review progress in developing nonviral lipid nanoparticle (LNP) delivery systems that have attractive properties, including ease of manufacture, reduced immune responses, multidosing capabilities, larger payloads, and flexibility of design. LNP systems represent the most advanced delivery systems for genetic drugs as it is expected that an LNP-short interfering RNA (siRNA) formulation will receive clinical approval from the Food and Drug Administration (FDA) in 2018 for treatment of the hereditary condition transthyretin-mediated amyloidosis, a fatal condition for which there is currently no treatment. This achievement is largely due to the development of optimized ionizable cationic lipids, arguably the most important factor in the clinical success of LNP-siRNA. In addition, we highlight potential LNP applications, including targeting tissues beyond the liver and therapeutic approaches based on messenger RNA or Clustered Regularly Interspaced Short Palindromic Repeats/Cas.
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