Abstract
The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.
Highlights
Gene therapy has gained considerable attention in the last few years for treating myriads of devastating diseases
[18,19], and polyethylene glycol (PEG)ylating cationic nature, liposomes bind non- to serum proteins and cause toxicity [14]. This has lipids [20,21] which tend to reduce the surface charge of cationic lipids, provide stability to lipid led to the use of helper lipids such as 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) [15], nanoparticles (LNPs), and reduce reticuloendothelial system (RES) uptake to increase their blood cholesterol (CHO) [16,17], phosphatidylcholines (PCs) [18,19], and polyethylene glycol (PEG)ylating circulation time [22]
The direct injection of nanoparticles in the spleen was conducted as it displays the same biodistribution of nanoparticles in the liver when compared with intravenous administration [92]
Summary
Gene therapy has gained considerable attention in the last few years for treating myriads of devastating diseases. Though many advances have been made in the field of gene therapy, the delivery of nucleic acid analogs (NAA), as well as genome editing proteins to the target site, continues to be an unresolved issue for the broad utility of gene therapy-based applications. This review exclusively summarizes a new generation of cationic lipids and cationic polymers and their conjugates for the delivery of nucleic acids, their analogs, as well as genome editing proteins for diverse therapeutic applications. 3. Spleen is the other organ other organ that constitutes MPS and causes accumulation as well as nanocarrier clearance [8]. 4. The that constitutes MPS and causes accumulation as well as nanocarrier clearance [8]. The lungs contribute toparticle the accumulation andtarget clearance of nanoparticles
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