Lipid-based nanoparticles and RNA as innovative neuro-therapeutics

Abstract

RNA-delivery is a promising tool to develop therapies for difficult to treat diseases such as neurological disorders, by silencing pathological genes or expressing therapeutic proteins. However, in many cases RNA delivery requires a vesicle that could effectively protect the molecule from bio-degradation, bypass barriers i.e., the blood brain barrier, transfer it to a targeted tissue and efficiently release the RNA inside the cells. Many vesicles such as viral vectors, and polymeric nanoparticles have been mentioned in literature. In this review, we focus in the discussion of lipid-based advanced RNA-delivery platforms. Liposomes and lipoplexes, solid lipid nanoparticles and lipid nanoparticles are the main categories of lipidic platforms for RNA-delivery to the central nervous systems (CNS). A variety of surface particles’ modifications and routes of administration have been studied to target CNS providing encouraging results in vivo. It is concluded that lipid-based nanoplatforms will play a key role in the development of RNA neuro-therapies.

Unraveling Therapeutic Opportunities and the Diagnostic Potential of microRNAs for Human Lung Cancer

“…They consist of a cationic lipid core and a polyethylene glycol (PEG) shell, which enhance their stability and reduce their immunogenicity [ 97 ]. Several preclinical studies have shown the efficacy of lipid-based nanoparticles in delivering miRNA therapeutics to lung cancer cells [ 98 , 99 ]. For example, a recent study demonstrated that the intravenous delivery of lipid-based nanoparticles containing miR-34a mimics can inhibit lung tumor growth and metastasis in a mouse model of NSCLC [ 100 ].…”

Use of microRNAs as Diagnostic, Prognostic, and Therapeutic Tools for Glioblastoma

“…Much hope is put into the development of lipid-based particles [ 181 ]. The main advantage of lipid-based particles is that, as they are naturally generated and can have natural tropisms for specific organs and even particular cell types, they can achieve the goal of being specific, potent, and safe [ 181 ]. In fact, mRNA vaccines are delivered in lipid-based particles and, while they are not yet very specific, they have been proven to be safe [ 182 ].…”

“…In fact, mRNA vaccines are delivered in lipid-based particles and, while they are not yet very specific, they have been proven to be safe [ 182 ]. Lipid-based particles can be generated artificially, which has the advantage of allowing a very homogeneous preparation, which is relatively easy to characterize, fabricate, store, and administrate [ 181 ]. Howevser, we still lack the knowledge to achieve extreme organ specificity with such types of particles.…”

Nanomaterials for mRNA‐based therapeutics: Challenges and opportunities

“…As intermediate tunneling, messenger ribonucleic acid (mRNA) can transport genetic codes from DNA to ribosomes for protein expression, which has emerged as a highly appealing potential to change the vaccination, protein replacement therapy, and other treatments for human diseases 1–4 . The first study of successful mRNA therapeutics in the preclinical application was published in 1990 5 .…”

“…As intermediate tunneling, messenger ribonucleic acid (mRNA) can transport genetic codes from DNA to ribosomes for protein expression, which has emerged as a highly appealing potential to change the vaccination, protein replacement therapy, and other treatments for human diseases. 1 , 2 , 3 , 4 The first study of successful mRNA therapeutics in the preclinical application was published in 1990. 5 In addition, due to the progress of mRNA manufacturing and intercellular delivery strategy, significant development and breakthrough have been made in the mRNA‐based therapy.…”