Microfluidic fabrication of lipid nanoparticles for the delivery of nucleic acids
[Display omitted] Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies. Viral vectors have been widely explored as a key platform for gene therapy due to their ability to efficiently transport nuc...
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| Published in | Advanced drug delivery reviews Vol. 184; p. 114197 |
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| Main Authors | , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
Netherlands
Elsevier B.V
01.05.2022
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| Subjects | |
| Online Access | Get full text |
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| Abstract | [Display omitted]
Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies. Viral vectors have been widely explored as a key platform for gene therapy due to their ability to efficiently transport nucleic acid-based therapeutics into the cells. However, the lack of precision in their delivery has led to several off-target toxicities. As such, various strategies in the form of non-viral gene delivery vehicles have been explored and are currenlty employed in several therapies including the SARS-CoV-2 vaccine. In this review, we discuss the opportunities lipid nanoparticles (LNPs) present for efficient gene delivery. We also discuss various synthesis strategies via microfluidics for high throughput fabrication of non-viral gene delivery vehicles. We conclude with the recent applications and clinical trials of these vehicles for the delivery of different genetic materials such as CRISPR editors and RNA for different medical conditions ranging from cancer to rare diseases. |
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| AbstractList | Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would otherwise not be possible with traditional therapies. Due to their ability to transport genomes to cells, Viral vectors have been a platform of choice in gene delivery applications. However, since their delivery is not precision based, the application has led to off-target toxicities. As such, various strategies in the form of non-viral gene delivery vehicles have been explored and are being developed. In this review, we discuss the opportunities lipid nanoparticles (LNPs) present for gene delivery, efficiently and precisely. We also discuss synthesis strategies via microfluidics used for high throughput fabrication of such non-viral gene delivery vehicles. Finally, the application of these vehicles for the delivery of different genetic materials such as peptides and RNA for different diseases ranging from more common diseases to rare diseases are explored. Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies. Viral vectors have been widely explored as a key platform for gene therapy due to their ability to efficiently transport nucleic acid-based therapeutics into the cells. However, the lack of precision in their delivery has led to several off-target toxicities. As such, various strategies in the form of non-viral gene delivery vehicles have been explored and are currenlty employed in several therapies including the SARS-CoV-2 vaccine. In this review, we discuss the opportunities lipid nanoparticles (LNPs) present for efficient gene delivery. We also discuss various synthesis strategies via microfluidics for high throughput fabrication of non-viral gene delivery vehicles. We conclude with the recent applications and clinical trials of these vehicles for the delivery of different genetic materials such as CRISPR editors and RNA for different medical conditions ranging from cancer to rare diseases.Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies. Viral vectors have been widely explored as a key platform for gene therapy due to their ability to efficiently transport nucleic acid-based therapeutics into the cells. However, the lack of precision in their delivery has led to several off-target toxicities. As such, various strategies in the form of non-viral gene delivery vehicles have been explored and are currenlty employed in several therapies including the SARS-CoV-2 vaccine. In this review, we discuss the opportunities lipid nanoparticles (LNPs) present for efficient gene delivery. We also discuss various synthesis strategies via microfluidics for high throughput fabrication of non-viral gene delivery vehicles. We conclude with the recent applications and clinical trials of these vehicles for the delivery of different genetic materials such as CRISPR editors and RNA for different medical conditions ranging from cancer to rare diseases. [Display omitted] Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies. Viral vectors have been widely explored as a key platform for gene therapy due to their ability to efficiently transport nucleic acid-based therapeutics into the cells. However, the lack of precision in their delivery has led to several off-target toxicities. As such, various strategies in the form of non-viral gene delivery vehicles have been explored and are currenlty employed in several therapies including the SARS-CoV-2 vaccine. In this review, we discuss the opportunities lipid nanoparticles (LNPs) present for efficient gene delivery. We also discuss various synthesis strategies via microfluidics for high throughput fabrication of non-viral gene delivery vehicles. We conclude with the recent applications and clinical trials of these vehicles for the delivery of different genetic materials such as CRISPR editors and RNA for different medical conditions ranging from cancer to rare diseases. Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies. Viral vectors have been widely explored as a key platform for gene therapy due to their ability to efficiently transport nucleic acid-based therapeutics into the cells. However, the lack of precision in their delivery has led to several off-target toxicities. As such, various strategies in the form of non-viral gene delivery vehicles have been explored and are currenlty employed in several therapies including the SARS-CoV-2 vaccine. In this review, we discuss the opportunities lipid nanoparticles (LNPs) present for efficient gene delivery. We also discuss various synthesis strategies via microfluidics for high throughput fabrication of non-viral gene delivery vehicles. We conclude with the recent applications and clinical trials of these vehicles for the delivery of different genetic materials such as CRISPR editors and RNA for different medical conditions ranging from cancer to rare diseases. |
| ArticleNumber | 114197 |
| Author | Willemen, Niels Prakash, Gyan Shokr, Ahmed Hassan, Shabir Bashir, Showkeen Muzamil Shin, Su Ryon |
| AuthorAffiliation | 1. Department of Molecular Metabolism, Harvard T. H. Chan School of Public Health, Boston, MA 02115 – USA 2 Division of Engineering in Medicine, Department of Medicine, Brigham and Women’s Hospital and Harvard Medical School, Cambridge MA 02139 – USA 5. Department of Biology, Khalifa University, Abu Dhabi, P.O 127788, United Arab Emirates 3. Department of Developmental BioEngineering, Faculty of Science and Technology, Technical Medical Centre, University of Twente, Drienerlolaan 5, 7522 NB, Enschede, The Netherlands 4. Biochemistry & Molecular Biology Lab, Division of Veterinary Biochemistry, Faculty of Veterinary Sciences and Animal Husbandry, Sher-e-Kashmir University of Agricultural Sciences and Technology, Srinagar -190006, Jammu and Kashmir, India |
| AuthorAffiliation_xml | – name: 3. Department of Developmental BioEngineering, Faculty of Science and Technology, Technical Medical Centre, University of Twente, Drienerlolaan 5, 7522 NB, Enschede, The Netherlands – name: 4. Biochemistry & Molecular Biology Lab, Division of Veterinary Biochemistry, Faculty of Veterinary Sciences and Animal Husbandry, Sher-e-Kashmir University of Agricultural Sciences and Technology, Srinagar -190006, Jammu and Kashmir, India – name: 1. Department of Molecular Metabolism, Harvard T. H. Chan School of Public Health, Boston, MA 02115 – USA – name: 5. Department of Biology, Khalifa University, Abu Dhabi, P.O 127788, United Arab Emirates – name: 2 Division of Engineering in Medicine, Department of Medicine, Brigham and Women’s Hospital and Harvard Medical School, Cambridge MA 02139 – USA |
| Author_xml | – sequence: 1 givenname: Gyan orcidid: 0000-0003-4725-9178 surname: Prakash fullname: Prakash, Gyan organization: Department of Molecular Metabolism, Harvard T. H. Chan School of Public Health, Boston, MA 02115, USA – sequence: 2 givenname: Ahmed surname: Shokr fullname: Shokr, Ahmed organization: Division of Engineering in Medicine, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Cambridge, MA 02139, USA – sequence: 3 givenname: Niels orcidid: 0000-0003-1061-3313 surname: Willemen fullname: Willemen, Niels organization: Department of Developmental BioEngineering, Faculty of Science and Technology, Technical Medical Centre, University of Twente, Drienerlolaan 5, 7522 NB Enschede, the Netherlands – sequence: 4 givenname: Showkeen Muzamil surname: Bashir fullname: Bashir, Showkeen Muzamil organization: Biochemistry & Molecular Biology Lab, Division of Veterinary Biochemistry, Faculty of Veterinary Sciences and Animal Husbandry, Sher-e-Kashmir University of Agricultural Sciences and Technology, Srinagar 190006, Jammu and Kashmir, India – sequence: 5 givenname: Su Ryon surname: Shin fullname: Shin, Su Ryon email: sshin4@bwh.harvard.edu organization: Division of Engineering in Medicine, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Cambridge, MA 02139, USA – sequence: 6 givenname: Shabir surname: Hassan fullname: Hassan, Shabir email: shassan@bwh.harvard.edu, shassan@bwh.harvard.edu organization: Division of Engineering in Medicine, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Cambridge, MA 02139, USA |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/35288219$$D View this record in MEDLINE/PubMed |
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| Keywords | Gene delivery CRISPR-delivery Non-viral vectors Lipid nanoparticles Microfluidics |
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Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with... Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would not have been possible with traditional therapies.... Gene therapy has emerged as a potential platform for treating several dreaded and rare diseases that would otherwise not be possible with traditional... |
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| StartPage | 114197 |
| SubjectTerms | COVID-19 COVID-19 Vaccines CRISPR-delivery Gene delivery Humans Lipid nanoparticles Lipids Liposomes Microfluidics Nanoparticles Non-viral vectors Nucleic Acids Rare Diseases SARS-CoV-2 |
| Title | Microfluidic fabrication of lipid nanoparticles for the delivery of nucleic acids |
| URI | https://dx.doi.org/10.1016/j.addr.2022.114197 https://www.ncbi.nlm.nih.gov/pubmed/35288219 https://www.proquest.com/docview/2639222928 https://pubmed.ncbi.nlm.nih.gov/PMC9035142 |
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