Liposomal steroid nano-drug is superior to steroids as-is in mdx mouse model of Duchenne muscular dystrophy.

Autor: Turjeman K; Laboratory of Membrane and Liposome Research, Department of Biochemistry and Molecular Biology, Institute for Medical Research Israel-Canada (IMRIC), The Hebrew University-Hadassah Medical School, Jerusalem, Israel., Yanay N; Pediatric Neuromuscular Laboratory, Hadassah Hebrew University Medical Center, Jerusalem, Israel; Felsenstein Medical Research Center (FMRC), Schneider Children's Medical Center, Tel-Aviv University, Israel., Elbaz M; Pediatric Neuromuscular Laboratory, Hadassah Hebrew University Medical Center, Jerusalem, Israel., Bavli Y; Laboratory of Membrane and Liposome Research, Department of Biochemistry and Molecular Biology, Institute for Medical Research Israel-Canada (IMRIC), The Hebrew University-Hadassah Medical School, Jerusalem, Israel., Gross M; Laboratory of Membrane and Liposome Research, Department of Biochemistry and Molecular Biology, Institute for Medical Research Israel-Canada (IMRIC), The Hebrew University-Hadassah Medical School, Jerusalem, Israel., Rabie M; Pediatric Neuromuscular Laboratory, Hadassah Hebrew University Medical Center, Jerusalem, Israel; Felsenstein Medical Research Center (FMRC), Schneider Children's Medical Center, Tel-Aviv University, Israel., Barenholz Y; Laboratory of Membrane and Liposome Research, Department of Biochemistry and Molecular Biology, Institute for Medical Research Israel-Canada (IMRIC), The Hebrew University-Hadassah Medical School, Jerusalem, Israel., Nevo Y; Pediatric Neuromuscular Laboratory, Hadassah Hebrew University Medical Center, Jerusalem, Israel; Felsenstein Medical Research Center (FMRC), Schneider Children's Medical Center, Tel-Aviv University, Israel. Electronic address: yoramne@clalit.org.il.
Jazyk: angličtina
Zdroj: Nanomedicine : nanotechnology, biology, and medicine [Nanomedicine] 2019 Feb; Vol. 16, pp. 34-44. Date of Electronic Publication: 2018 Dec 07.
DOI: 10.1016/j.nano.2018.11.012
Abstrakt: Glucocorticosteroids are the most efficacious anti-inflammatory agents and the gold standard treatment in Duchenne muscular dystrophy (DMD). However, their chronic use may lead to severe side effects. We evaluated the use of a novel injectable steroidal nano-drug in mdx mouse model of DMD by comparing the efficacy of nano-liposomes remotely loaded with the steroid prodrug, methylprednisolone hemisuccinate (MPS) with the same steroid as-is, in short (4-weeks) and long-term (58-weeks) treatments. Liposomal-MPS was selectively targeted to the mouse diaphragm, the most dystrophic muscle at early stage of the disease. The bioactivity of the steroidal nano-drug was evidenced by a significant decreased serum TGF-β and reduced diaphragm macrophage infiltration after short-term treatment. In the long-term, the treatment with liposomal-MPS not only demonstrated improved muscle strength and mobility it also induced lower tibia and lumbar vertebrae osteoporosis indicating much lower bone related adverse effects.
(Copyright © 2018 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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