Platform for the Delivery of Unformulated RNA In Vivo.

Animals Drug Delivery Systems Liposomes Mice Nanoparticles RNA, Small Interfering / genetics
Antisense therapy Drug delivery Inflammatory bowel disease Mucosal delivery RNA Sjögren syndrome Antigen B Ulcerative colitis Ultrasound β-catenin

Journal

Journal of pharmaceutical sciences
ISSN: 1520-6017
Titre abrégé: J Pharm Sci
Pays: United States
ID NLM: 2985195R

Informations de publication

Date de publication:
06 2022
Historique:
received: 12 10 2021
revised: 07 12 2021
accepted: 08 12 2021
pubmed: 16 12 2021
medline: 25 5 2022
entrez: 15 12 2021
Statut: ppublish

Résumé

The successful delivery of RNA therapeutics is the gating hurdle to greater clinical translation and utility of this novel class of therapeutics. Delivery strategies today are limited and predominantly rely on lipid nanoparticles or conjugates, which can facilitate hepatic delivery but are poor for achieving uptake outside the liver. The ability to deliver RNA to other organs outside the liver in a formulation-agnostic approach could serve to unlock the broader potential of these therapies and enable their use in a broader set of disease. Here we demonstrate this formulation-agnostic delivery of two model siRNAs using low-frequency ultrasound to the gastrointestinal (GI) tract. Unformulated siRNAs targeting β-catenin (Ctnnb 1) and Sjögren syndrome antigen B (SSB) genes were successfully delivered to colonic mucosa in mice, achieving robust knockdown of the target mRNA from whole-colon tissue samples. Indeed, the capacity to target and successfully suppress expression from genes underscores the power of this platform to rapidly deliver unformulated and unoptimized sequences against a range of targets in the GI tract.

Identifiants

DOI: 10.1016/j.xphs.2021.12.008 PMID: 34906584
pubmed: 34906584
pii: S0022-3549(21)00681-X
doi: 10.1016/j.xphs.2021.12.008
pii:
doi:

Substances chimiques

Lipid Nanoparticles 0
Liposomes 0
RNA, Small Interfering 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

1770-1775

Informations de copyright

Copyright © 2021 American Pharmacists Association. Published by Elsevier Inc. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interests M.M.F., T.d.R., H.T., E.R., and C.M.S. were employees of Suono Bio, Inc. at the time of this work. R.L., G.T., and C.M.S. are co-founders of Suono Bio, Inc., and co-inventors on multiple patent applications on the topic of ultrasound-mediated drug delivery. R.L. has a financial interest in companies such as Alnylam and Moderna, which focus on developing RNA-based therapeutics. Complete details of all relationships for profit and not for profit for G.T. can be found at the following link: https://www.dropbox.com/sh/szi7vnr4a2ajb56/AABs5N5i0q9AfT1IqIJAE-T5a?dl=0. Complete details for R.L. can be found at the following link: https://www.dropbox.com/s/yc3xqb5s8s94v7x/Rev%20Langer%20COI.pdf?dl=0

Auteurs

Marion M France (MM)

Suono Bio, Inc., 200 Foxborough Blvd., Suite 100, Foxborough, MA 02035, USA.

Tony Del Rio (TD)

Suono Bio, Inc., 200 Foxborough Blvd., Suite 100, Foxborough, MA 02035, USA.

Hannah Travers (H)

Suono Bio, Inc., 200 Foxborough Blvd., Suite 100, Foxborough, MA 02035, USA; Northeastern University, 360 Huntington Ave., Boston, MA 02115, USA.

Erin Raftery (E)

Northeastern University, 360 Huntington Ave., Boston, MA 02115, USA.

Robert Langer (R)

David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, 500 Main St., Cambridge, MA 02139, USA.

Giovanni Traverso (G)

Division of Gastroenterology, Brigham and Women's Hospital, 65 Landsdowne St., Suite 252, Cambridge, MA 02139, USA; Harvard Medical School, Boston, MA 02115, USA; Department of Mechanical Engineering, Massachusetts Institute of Technology, 77 Massachusetts Ave., Cambridge, MA 02139, USA.

Carl M Schoellhammer (CM)

Suono Bio, Inc., 200 Foxborough Blvd., Suite 100, Foxborough, MA 02035, USA. Electronic address: cms@suonobio.com.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Platform for the Delivery of Unformulated RNA In Vivo.
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Systèmes de délivrance de médicaments › Platform for the Delivery of Unformulated RNA In Vivo.
questionsmedicales.fr › Technologie, industrie et agriculture › Produits manufacturés › Matériaux biomimétiques › Membrane artificielle › Liposomes › Platform for the Delivery of Unformulated RNA In Vivo.
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Platform for the Delivery of Unformulated RNA In Vivo.
questionsmedicales.fr › Technologie, industrie et agriculture › Produits manufacturés › Nanostructures › Nanoparticules › Platform for the Delivery of Unformulated RNA In Vivo.
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Acides nucléiques › ARN › ARN non traduit › Petit ARN non traduit › Petit ARN interférent › Platform for the Delivery of Unformulated RNA In Vivo.