Systemic modulation of lymphocyte subsets using siRNAs delivered via targeted lipid nanoparticles

Inbal Hazan-Halevy, Daniel Rosenblum, Srinivas Ramishetti, Dan Peer*

*Corresponding author for this work

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

4 Scopus citations

Abstract

Systemic delivery of RNA interference (RNAi) payloads for manipulation of gene expression in lymphocytes holds a great potential as a novel therapeutic modality for hematological malignancies and autoimmune disorders. However, lymphocytes are among the most difficult cells to transfect with RNAi, as they are resistant to conventional transfection reagents and are dispersed throughout the body, making it a challenge to successfully deliver these payloads via systemic administration route. We have developed a strategy to target lymphocytes and deliver RNAi payloads in a cell-specific manner to induce therapeutic gene silencing. This approach utilizes antibodies that decorate lipid nanoparticle surfaces to home into lymphocyte subsets. This approach opens new avenues for discovery of new drug targets and potentially for therapeutics.

Original languageEnglish
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages151-159
Number of pages9
DOIs
StatePublished - 2019

Publication series

NameMethods in Molecular Biology
Volume1974
ISSN (Print)1064-3745
ISSN (Electronic)1940-6029

Funding

FundersFunder number
Leona M. and Harry B. Helmsley Charitable Trust
Tel Aviv University
Varda and Boaz Dotan Research Center for Hemato-Oncology Research, Tel Aviv University

    Keywords

    • Gene silencing
    • Lipid nanoparticles (LNPs)
    • RNA interference (RNAi)
    • Small interfering RNAs (siRNAs)

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