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Lipid nanoparticle-mediated drug delivery to the brain
Adv Drug Deliv Rev. 2023 May 5:114861. doi: 10.1016/j.addr.2023.114861. Online ahead of print.ABSTRACTLipid nanoparticles (LNPs) have revolutionized the field of drug delivery through their applications in siRNA delivery to the liver (Onpattro) and their use in the Pfizer-BioNTech and Moderna COVID-19 mRNA vaccines. While LNPs have been extensively studied for the delivery of RNA drugs to muscle and liver targets, their potential to deliver drugs to challenging tissue targets such as the brain remains underexplored. Multiple brain disorders currently lack safe and effective therapies and therefore repurposing LNPs could po...
Source: Advanced Drug Delivery Reviews - May 7, 2023 Category: Drugs & Pharmacology Authors: Purva Khare Sara X Edgecomb Christine M Hamadani Eden E L Tanner Devika S Manickam Source Type: research

The role of lipid components in lipid nanoparticles for vaccines and gene therapy
Adv Drug Deliv Rev. 2022 Jul 1:114416. doi: 10.1016/j.addr.2022.114416. Online ahead of print.ABSTRACTLipid nanoparticles (LNPs) play a key role in mRNA vaccines against COVID-19. In addition, many preclinical and clinical studies, including the siRNA-LNP product, Onpattro®, highlight that LNPs unlock the potential of nucleic acid-based therapies and vaccines. To understand how this 'key' works, we need to learn about the building blocks that constitute LNPs. In this review, we discuss what each lipid component adds to the LNP delivery platform in terms of size, structure, stability, apparent pKa, nucleic acid encapsulati...
Source: Advanced Drug Delivery Reviews - July 5, 2022 Category: Drugs & Pharmacology Authors: Camilla Hald Albertsen Jayesh Kulkarni Dominik Witzigmann Marianne Lind Karsten Petersson Jens B Simonsen Source Type: research

Regulatory guidelines and preclinical tools to study the biodistribution of RNA therapeutics
Adv Drug Deliv Rev. 2022 Mar 26;184:114236. doi: 10.1016/j.addr.2022.114236. Online ahead of print.ABSTRACTThe success of the messenger RNA-based COVID-19 vaccines of Moderna and Pfizer/BioNTech marks the beginning of a new chapter in modern medicine. However, the rapid rise of mRNA therapeutics has resulted in a regulatory framework that is somewhat lagging. The current guidelines either do not apply, do not mention RNA therapeutics, or do not have widely accepted definitions. This review describes the guidelines for preclinical biodistribution studies of mRNA/siRNA therapeutics and highlights the relevant differences for...
Source: Advanced Drug Delivery Reviews - March 30, 2022 Category: Drugs & Pharmacology Authors: P Vervaeke S E Borgos N N Sanders F Combes Source Type: research