Lipoplex-based Therapeutics for Effective Oligonucleotide Delivery: A Compendious Review


Authors: P.P. Singh, V. Vithalapuram, S. Metre and R. Kodipyaka

Journal: Journal of Liposome Research

DOI: 10.1080/08982104.2019.1652645

Publication - Abstract

August 19, 2019

Abstract

Oligonucleotide is emerging as a novel class of therapeutics due to its high specificity, and ability to manage the incorrigible diseases with targeted action. The expedited growth evident from past few decades unveils the potentiality of the oligonucleotide as the future medicine. Various studies based on the vector-based system have shown the superiority of the lipid over other non-viral vectors. The lipid-based systems are most often exploited for the development of safe and efficient gene delivery systems. The factors influencing the structure, stability, internalization, and transfection of the lipoplex required for effective lipoplex delivery system are being explored. Furthermore, safety considerations and the status of lipoplex clinical trials are also addressed. The need of more scalable methods, which can be applied at industrial level, is identified and exploited. The aim is to manufacture products with long-term shelf life to support clinical trials and their subsequent market use. The design of lipoplexes leads to efficient entrapment of nucleic acid with enhanced in-vitro and in-vivo milieu stability, facilitating uptake and cellular targeting. The increasing number of entry of lipoplex-based gene therapy in the clinical trials shows the potential of lipoplex as a well-characterized system with consistent quality and reliable performance. The concomitant development of novel lipids, enhanced understanding of the uptake mechanism and formulation design characteristics have increased lipoplex-based oligonucleotide delivery system access to and success in the clinical trials and regulatory nod.

Advanced Search

close
  • Publications
  • Application Notes
  • Posters
  • Workshops
  • Videos & Webinars
  • Blog Posts
Search

Browse by Category

  • Application
    • Diagnostic and Imaging
    • Genetic Medicine
    • Hematology
    • Metabolic Disorders
    • Neuroscience
    • Oncology
    • Skeletal Disorders
    • Targeted Drug Delivery
    • Vaccines
    • Other Applications
  • Formulation
    • Liposomes
    • Nucleic Acid Lipid Nanoparticles
    • Polymeric Nanoparticles
    • Other Formulations
  • Payload
    • DNA
    • microRNA
    • mRNA
    • siRNA
    • Small Molecule Drugs
    • Other Payloads


related content

Publication - Summary

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

J. Finn, A. Smith, M. Patel, L. Shaw, M. Youniss, J. Heteren, T. Dirstine, C. Ciullo, R. Lescarbeau, J. Seitzer, R. Shah, A. Shah, D. Ling, J. Growe, M. Pink, E. Rohde, K. Wood, W. Salomon, W. Harrington, C. Dombrowski, W. Strapps, Y. Chang, D. Morrissey

Hereditary transthryretin amyloidosis is a rare disease caused by mutations in the gene encoding the protein transthyretin (TTR), causing it to misfold into amyloid plaques, leading to debilitating symptoms. In 2018,...

Read More


Publication - Abstract

Within this paper we present work that has the ability to de-risk the translation of liposomes from bench to the clinic. We have used microfluidics for the rapid and scale-independent manufacture of liposomes and have incorporated in-line purification and at-line monitoring of pa...
Read More


Stay Informed

Sign up today to automatically receive new Precision NanoSystems application notes, conference posters, relevant science publications, and webinar invites.

MENU
菜单
X
X