TY - CHAP
T1 - Systemic modulation of lymphocyte subsets using siRNAs delivered via targeted lipid nanoparticles
AU - Hazan-Halevy, Inbal
AU - Rosenblum, Daniel
AU - Ramishetti, Srinivas
AU - Peer, Dan
N1 - Publisher Copyright:
© Springer Science+Business Media, LLC, part of Springer Nature 2019.
PY - 2019
Y1 - 2019
N2 - 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.
AB - 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.
KW - Gene silencing
KW - Lipid nanoparticles (LNPs)
KW - RNA interference (RNAi)
KW - Small interfering RNAs (siRNAs)
UR - http://www.scopus.com/inward/record.url?scp=85066112184&partnerID=8YFLogxK
U2 - 10.1007/978-1-4939-9220-1_11
DO - 10.1007/978-1-4939-9220-1_11
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C2 - 31099001
AN - SCOPUS:85066112184
T3 - Methods in Molecular Biology
SP - 151
EP - 159
BT - Methods in Molecular Biology
PB - Humana Press Inc.
ER -