Stem Cell Therapy as a Treatment for Osteogenesis Imperfecta

被引:34
|
作者
Gotherstrom, Cecilia [1 ]
Walther-Jallow, Lilian [1 ]
机构
[1] Karolinska Inst, Dept Clin Sci Intervent & Technol, Div Obstet & Gynecol, ANA Futura, Floor 8,Alfred Nobels Alle 8, S-14152 Stockholm, Sweden
基金
欧盟地平线“2020”; 瑞典研究理事会;
关键词
Osteogenesis imperfecta; Brittle bones; Stem cell therapy; Stem cell transplantation; Prenatal therapy; Mesenchymal stem cells; MESENCHYMAL STROMAL CELLS; IN-UTERO TRANSPLANTATION; INTRAUTERINE TRANSPLANTATION; IMMUNOLOGICAL-PROPERTIES; MUSCLE DIFFERENTIATION; FETAL; BONE; CLASSIFICATION; PHENOTYPE; RESPONSES;
D O I
10.1007/s11914-020-00594-3
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Purpose of Review Osteogenesis imperfecta (OI) is a chronic disease with few treatment options available. The purpose of this review is to provide an overview on treating OI with mesenchymal stem cells (MSC). Recent Findings Off-the-shelf MSC have a good safety profile and exhibit multilineage differentiation potential and a low immunogenic profile and are easy to manufacture. Their ability to migrate, engraft, and differentiate into bone cells, and also to act via paracrine effects on the recipient's tissues, makes MSC candidates as a clinical therapy for OI. Due to their high osteogenic potency, fetal MSC offer an even higher therapeutic potential in OI compared with MSC derived from adult sources. Preclinical and initial clinical data support the use of MSC in treating OI. The characteristics of MSC make them of great interest in treating OI. MSC may be safely transplanted via intravenous administration and show potential positive clinical effects.
引用
收藏
页码:337 / 343
页数:7
相关论文
共 50 条
  • [31] Classification of Osteogenesis Imperfecta revisited
    Van Dijk, F. S.
    Pals, G.
    Van Rijn, R. R.
    Nikkels, P. G. J.
    Cobben, J. M.
    EUROPEAN JOURNAL OF MEDICAL GENETICS, 2010, 53 (01) : 1 - 5
  • [32] Circulating TGF-β Pathway in Osteogenesis Imperfecta Pediatric Patients Subjected to MSCs-Based Cell Therapy
    Infante, Arantza
    Cabodevilla, Leire
    Gener, Blanca
    Rodriguez, Clara I.
    FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY, 2022, 10
  • [33] Gene therapy approaches for osteogenesis imperfecta
    Niyibizi, C
    Wang, S
    Mi, Z
    Robbins, PD
    GENE THERAPY, 2004, 11 (04) : 408 - 416
  • [34] Gene therapy approaches for osteogenesis imperfecta
    C Niyibizi
    S Wang
    Z Mi
    PD Robbins
    Gene Therapy, 2004, 11 : 408 - 416
  • [35] Pathophysiology and Therapy for Patients with Osteogenesis imperfecta
    Semler, O.
    Rehberg, M.
    Hoyer-Kuhn, H.
    OSTEOLOGIE, 2018, 27 (04) : 194 - 199
  • [36] Therapy with pamidronate in children with osteogenesis imperfecta
    Marginean, Otilia
    Tamasanu, Raluca Corina
    Mang, Niculina
    Mozos, Ioana
    Brad, Giorgiana Flavia
    DRUG DESIGN DEVELOPMENT AND THERAPY, 2017, 11 : 2507 - 2515
  • [37] Bisphosphonate therapy for severe osteogenesis imperfecta
    Glorieux, FH
    JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM, 2000, 13 : 989 - 992
  • [38] Evaluation of a Modified Pamidronate Protocol for the Treatment of Osteogenesis Imperfecta
    Palomo, Telma
    Andrade, Maria C.
    Peters, Barbara S. E.
    Reis, Fernanda A.
    Carvalhaes, Joao Tomas A.
    Glorieux, Francis H.
    Rauch, Frank
    Lazaretti-Castro, Marise
    CALCIFIED TISSUE INTERNATIONAL, 2016, 98 (01) : 42 - 48
  • [39] Fractures in Osteogenesis Imperfecta: Pathogenesis, Treatment, Rehabilitation and Prevention
    Nijhuis, Wouter
    Verhoef, Marjolein
    van Bergen, Christiaan
    Weinans, Harrie
    Sakkers, Ralph
    CHILDREN-BASEL, 2022, 9 (02):
  • [40] Osteogenesis imperfecta: pathophysiology and treatment; [Osteogenesis imperfecta: Update zu Pathophysiology und Therapie]
    Hoyer-Kuhn H.
    Netzer C.
    Semler O.
    Wiener Medizinische Wochenschrift, 2015, 165 (13-14) : 278 - 284