Bone-Specific Drug Delivery for Osteoporosis and Rare Skeletal Disorders
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REGENERATIVE BIOLOGY AND MEDICINE IN OSTEOPOROSIS (S BRYANT AND M KREBS, SECTION EDITORS)
Bone-Specific Drug Delivery for Osteoporosis and Rare Skeletal Disorders Kazuki Sawamoto 1 & J. Víctor Álvarez 1 & Angélica María Herreño 1 & Francisco J. Otero-Espinar 2 & Maria L. Couce 3 & Carlos J. Alméciga-Díaz 4 & Shunji Tomatsu 1,5,6,7
# Springer Science+Business Media, LLC, part of Springer Nature 2020
Abstract Purpose of Review The skeletal system provides an important role to support body structure and protect organs. The complexity of its architecture and components makes it challenging to deliver the right amount of the drug into bone regions, particularly avascular cartilage lesions. In this review, we describe the recent advance of bone-targeting methods using bisphosphonates, polymeric oligopeptides, and nanoparticles on osteoporosis and rare skeletal diseases. Recent Findings Hydroxyapatite (HA), a calcium phosphate with the formula Ca10(PO4)6(OH)2, is a primary matrix of bone mineral that includes a high concentration of positively charged calcium ion and is found only in the bone. This unique feature makes HA a general targeting moiety to the entire skeletal system. We have applied bone-targeting strategy using acidic amino acid oligopeptides into lysosomal enzymes, demonstrating the effects of bone-targeting enzyme replacement therapy and gene therapy on bone and cartilage lesions in inherited skeletal disorders. Virus or no-virus gene therapy using techniques of engineered capsid or nanomedicine has been studied preclinically for skeletal diseases. Summary Efficient drug delivery into bone lesions remains an unmet challenge in clinical practice. Bone-targeting therapies based on gene transfer can be potential as new candidates for skeletal diseases. Keywords Bone-targeting . Osteoporosis . Metabolic rare skeletal disorders . Acidic amino acid oligopeptide . Nanoparticles
Abbreviations AAV Adeno-associated virus ADL Activity of daily living ALN Alendronate
Asp BMD BPs C6S
Aspartic acid Bone mineral density Bisphosphonates Chondroitin-6-sulfate
This article is part of the Topical Collection on Regenerative Biology and Medicine in Osteoporosis Kazuki Sawamoto, J. Víctor Álvarez1, and Angélica María Herreño are joint first authors * Shunji Tomatsu [email protected]
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Institute for the Study of Inborn Errors of Metabolism, Faculty of Science, Pontificia Universidad Javeriana, Bogotá DC, Colombia
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Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA
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Department of Pediatrics, Graduate School of Medicine, Gifu University, Gifu, Japan
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Department of Pharmacology, Pharmacy and Pharmaceutical Technology, School of Pharmacy, University of Santiago de Compostela, Santiago de Compostela, Spain
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Department of Pediatrics, Thomas Jefferson University, Philadelphia, PA, USA
Department of CC Foren. An. Pat, Gin. and Obst. and Paed. Neonatology Service, Metabolic Unit, University Clinic Hospital of Santiago de Compostela, Santiago de Compostela, Spain
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Department of Biomedical Resear
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