Iron Oxide Nanoparticle-Mediated siRNA Delivery System for Huntington´s Disease Treatment

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F61388980%3A_____%2F23%3A00572491" target="_blank" >RIV/61388980:_____/23:00572491 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/61389013:_____/23:00572491 RIV/67985904:_____/23:00572491

Výsledek na webu

<a href="https://asep.lib.cas.cz/arl-cav/cs/csg/?repo=crepo1&key=41122578716" target="_blank" >https://asep.lib.cas.cz/arl-cav/cs/csg/?repo=crepo1&key=41122578716</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1021/acsanm.2c03936" target="_blank" >10.1021/acsanm.2c03936</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Iron Oxide Nanoparticle-Mediated siRNA Delivery System for Huntington´s Disease Treatment

Popis výsledku v původním jazyce

Huntington's disease (HD) is an autosomal dominant disease affecting neurons predominantly in the striatum due to the production of the toxic huntingtin protein. Lowering the concentration of mutant huntingtin is a promising therapeutic approach, and a suitable delivery system is fascinating. Nanoparticles (NPs) minimize the host immune response and have no limit concerning the number of NPs administered. They are safe, targeted, and effective for RNA therapeutics providing a significant mode to cross the blood-brain barrier for a broad range of clinical applications. The present study generated and characterized magnetic NPs (MNPs) using the co-precipitation method with a mean particle size of around 10-20 nm. The dynamic light scattering and zeta potential measurements showed that NPs exhibited narrow size distribution and sufficient colloidal stability. These oleic acid-coated MNPs were further cross-linked with polyethyleneimine and designed to deliver interfering RNA into human embryonic kidney cells (HEK-293) driven by an external magnetic field. These MNPs showed low cytotoxicity with high transfection efficiency. Furthermore, a transient downregulation was observed in endogenous huntingtin protein obtained by RT-PCR and Western blot analysis. Thus, these MNPs may represent a promising and efficient platform for siRNA delivery and provide a potential treatment strategy for HD.

Název v anglickém jazyce

Iron Oxide Nanoparticle-Mediated siRNA Delivery System for Huntington´s Disease Treatment

Popis výsledku anglicky

Huntington's disease (HD) is an autosomal dominant disease affecting neurons predominantly in the striatum due to the production of the toxic huntingtin protein. Lowering the concentration of mutant huntingtin is a promising therapeutic approach, and a suitable delivery system is fascinating. Nanoparticles (NPs) minimize the host immune response and have no limit concerning the number of NPs administered. They are safe, targeted, and effective for RNA therapeutics providing a significant mode to cross the blood-brain barrier for a broad range of clinical applications. The present study generated and characterized magnetic NPs (MNPs) using the co-precipitation method with a mean particle size of around 10-20 nm. The dynamic light scattering and zeta potential measurements showed that NPs exhibited narrow size distribution and sufficient colloidal stability. These oleic acid-coated MNPs were further cross-linked with polyethyleneimine and designed to deliver interfering RNA into human embryonic kidney cells (HEK-293) driven by an external magnetic field. These MNPs showed low cytotoxicity with high transfection efficiency. Furthermore, a transient downregulation was observed in endogenous huntingtin protein obtained by RT-PCR and Western blot analysis. Thus, these MNPs may represent a promising and efficient platform for siRNA delivery and provide a potential treatment strategy for HD.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

10402 - Inorganic and nuclear chemistry

Projekt

Výsledek vznikl pri realizaci vícero projektů. Více informací v záložce Projekty.

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2023

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

ACS Applied Nano Materials

ISSN

2574-0970

e-ISSN

—

Svazek periodika

6

Číslo periodika v rámci svazku

7

Stát vydavatele periodika

US - Spojené státy americké

Počet stran výsledku

11

Strana od-do

5106-5116

Kód UT WoS článku

000959870100001

EID výsledku v databázi Scopus

2-s2.0-85151323256