Literature DB >> 26419658

RALA-mediated delivery of FKBPL nucleic acid therapeutics.

Rachel Bennett1, Anita Yakkundi1, Hayley D McKeen1, Lana McClements1, Thomas J McKeogh1, Cian M McCrudden1, Kenneth Arthur2, Tracy Robson1, Helen O McCarthy1.   

Abstract

AIMS: RALA is a novel 30 mer bioinspired amphipathic peptide that is showing promise for gene delivery. Here, we used RALA to deliver the FK506-binding protein like - FKBPL gene (pFKBPL) - a novel member of the immunophilin protein family. FKBPL is a secreted protein, with overexpression shown to inhibit angiogenesis, tumor growth and stemness, through a variety of intra- and extracellular signaling mechanisms. We also elucidated proangiogenic activity and stemness after utilizing RALA to deliver siRNA (siFKBPL). MATERIALS &
METHODS: The RALA/pFKBPL and RALA/siFKBPL nanoparticles were characterized in terms of size, charge, stability and toxicity. Overexpression and knockdown of FKBPL was assessed in vitro and in vivo.
RESULTS: RALA delivered both pFKBPL and siFKBPL with less cytotoxicity than commercially available counterparts. In vivo, RALA/pFKBPL delivery retarded tumor growth, and prolonged survival with an associated decrease in angiogenesis, while RALA/siFKBPL had no effect on tumor growth rate or survival, but resulted in an increase in angiogenesis and stemness.
CONCLUSION: RALA is an effective delivery system for both FKBPL DNA and RNAi and highlights an alternative therapeutic approach to harnessing FKBPL's antiangiogenic and antistemness activity.

Entities:  

Keywords:  FKBPL DNA; RALA peptide; RNAi; antiangiogenic; nanoparticle

Year:  2015        PMID: 26419658      PMCID: PMC4910961          DOI: 10.2217/nnm.15.115

Source DB:  PubMed          Journal:  Nanomedicine (Lond)        ISSN: 1743-5889            Impact factor:   6.096


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