Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders

Author:

Conejos-Sánchez I.12345ORCID,Gallon E.123,Niño-Pariente A.123ORCID,Smith J. A.45678,De la Fuente A. G.45679ORCID,Di Canio L.45679,Pluchino S.4567ORCID,Franklin R. J. M.45679,Vicent M. J.123ORCID

Affiliation:

1. Centro de Investigación Príncipe Felipe. Polymer Therapeutics Laboratory

2. 46012 Valencia

3. Spain

4. Dept. Clinical Neurosciences and NIHR Biomedical Research Centre

5. Clifford Allbutt Building-Cambridge Biosciences Campus

6. University of Cambridge

7. UK

8. Cambridge Innovation Technologies Consulting (CITC) Limited

9. Wellcome-Medical Research Council Cambridge Stem Cell Institute

Abstract

Novel biodegradable and biocompatible polyornithine derivatives as non-viral vectors for siRNA exhibit effectively silence gene expression in primary neural cells.

Funder

Ministerio de Ciencia e Innovación

Generalitat Valenciana

European Regional Development Fund

Publisher

Royal Society of Chemistry (RSC)

Subject

General Materials Science

Reference91 articles.

1. Current preclinical small interfering RNA (siRNA)-based conjugate systems for RNA therapeutics

2. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

3. Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

4. https://www.alnylam.com/alnylam-rnai-pipeline/clinical-trials/

5. http://investors.alnylam.com/news-releases/news-release-details/alnylam-submits-marketing-authorization-application-european

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