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Smart peptide nanoparticles for efficient and safe gene therapy
Third-party funded project
Project title Smart peptide nanoparticles for efficient and safe gene therapy
Principal Investigator(s) Palivan, Cornelia
Benenson, Yaakov
Project Members Tarvirdipour, Shabnam
Organisation / Research unit Departement Chemie / Physikalische Chemie (Palivan)
Project start 01.03.2017
Probable end 28.02.2021
Status Completed
Abstract

Gene therapy constitutes a promising novel modality for treatment of genetic diseases as well as cancer. However, widespread clinical adoption of gene therapies is still in its infancy not least due to various issues associated with using viral vectors to deliver transgenes to target tissues or tumors in patients. Therefore, non-viral delivery methods for DNA and possibly RNA could have advantages in terms of immunogenicity, toxicity, and more.
The aim of this project is to develop the next generation of non-viral DNA delivery tools using smart peptide nanoparticles specifically designed to carry theranostic agents comprising DNA and fluorescent molecules (as reporters), and to test them both in vitro and in vivo. We plan to implement extended nucleotide condensing sites of oligo histidines, and decrease the size of the self-assembled system below 50 nm.
Our study will provide a two-pronged strategy combining cutting-edge design of nanoscience based carriers with a suite of methods and assays that enable their detailed interrogation and understanding of their delivery mechanisms in vitro and in vivo. It will also form a basis for novel and expanded theranostic strategies due to co-entrapment of fluorescent molecules with large DNA payloads. This capability will, on one hand, enable the delivery of theranostic gene circuits; on the other hand, the entrapment of fluorescent dyes will enable facile readout of particle delivery in time and space that does not rely on gene expression. Importantly, this study takes advantage of established expertise and experimental capabilities in both collaborating labs.

Keywords peptide nanoparticles, gene therapy, DNA
Financed by Other sources

Published results ()

  ID Autor(en) Titel ISSN / ISBN Erschienen in Art der Publikation
4634461  Tarvirdipour, Shabnam; Skowicki, Michal; Schoenenberger, Cora-Ann; Palivan, Cornelia G.  Peptide-Assisted Nucleic Acid Delivery Systems on the Rise  1661-6596 ; 1422-0067  International Journal of Molecular Sciences  Publication: JournalArticle (Originalarbeit in einer wissenschaftlichen Zeitschrift) 
4607659  Tarvirdipour, Shabnam; Huang, Xinan; Mihali, Voichita; Schoenenberger, Cora-Ann; Palivan, Cornelia G.  Peptide-Based Nanoassemblies in Gene Therapy and Diagnosis: Paving the Way for Clinical Application  1420-3049  Molecules  Publication: JournalArticle (Originalarbeit in einer wissenschaftlichen Zeitschrift) 
4526062  Tarvirdipour, Shabnam; Schoenenberger, Cora-Ann; Benenson, Yaakov; Palivan, Cornelia G. G.  A self-assembling amphiphilic peptide nanoparticle for the efficient entrapment of DNA cargoes up to 100 nucleotides in length  1744-683X ; 1744-6848  Soft Matter  Publication: JournalArticle (Originalarbeit in einer wissenschaftlichen Zeitschrift) 
4646672  Tarvirdipour, Shabnam; Skowicki, Michal; Schoenenberger, Cora-Ann; Kapinos, Larisa E.; Lim, Roderick Y. H.; Benenson, Yaakov; Palivan, Cornelia G.  A self-assembling peptidic platform to boost the cellular uptake and nuclear delivery of oligonucleotides  2047-4830 ; 2047-4849  Biomaterials science  Publication: JournalArticle (Originalarbeit in einer wissenschaftlichen Zeitschrift) 
   

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29/03/2024