Modification of COL1A1 in Autologous Adipose Tissue-Derived Progenitor Cells Rescues the Bone Phenotype in a Mouse Model of Osteogenesis Imperfecta.
ADIPOSE-DERIVED MESENCHYMAL STEM CELLS
GENE MODIFICATION
HIPPO/YAP
OSTEOGENESIS IMPERFECTA
OSTEOGENIC DIFFERENTIATION
Journal
Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
ISSN: 1523-4681
Titre abrégé: J Bone Miner Res
Pays: United States
ID NLM: 8610640
Informations de publication
Date de publication:
08 2021
08 2021
Historique:
revised:
16
04
2021
received:
28
09
2020
accepted:
21
04
2021
pubmed:
6
5
2021
medline:
10
8
2021
entrez:
5
5
2021
Statut:
ppublish
Résumé
Osteogenesis imperfecta (OI) is a congenital genetic disorder mainly manifested as bone fragility and recurrent fracture. Mutation of COL1A1/COL1A2 genes encoding the type I collagen are most responsible for the clinical patients. Allogenic mesenchymal stem cells (MSCs) provide the potential to treat OI through differentiation into osteoblasts. Autologous defective MSCs have not been utilized in OI treatment mainly because of their impaired osteogenesis, but the latent mechanism has not been well understood. Here, the relative signaling abnormality of adipose-derived mesenchymal stem cells (ADSCs) isolated from OI type I mice (Col1a1
Substances chimiques
Collagen Type I
0
Collagen Type I, alpha 1 Chain
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1521-1534Informations de copyright
© 2021 American Society for Bone and Mineral Research (ASBMR).
Références
Marini JC, Forlino A, Bächinger HP, et al. Osteogenesis imperfecta. Nat Rev Dis Primers. 2017;3:17052.
Forlino A, Marini JC. Osteogenesis imperfecta. Lancet. 2016;387(10028):1657-1671.
Sillence DO, Rimoin DL, Danks DM. Clinical variability in osteogenesis imperfecta-variable expressivity or genetic heterogeneity. Birth Defects Orig Artic Ser. 1979;15(5B):113-129.
Golshani KR, Ludwig MR, Cohn PL, et al. Osteogenesis imperfecta. Del Med J. 2016;88(6):178-185.
Trejo P, Fassier F, Glorieux FH, et al. Diaphyseal femur fractures in osteogenesis imperfecta: characteristics and relationship with bisphosphonate treatment. J Bone Miner Res. 2017;32(5):1034-1039.
Rijks EB, Bongers BC, Vlemmix MJ, et al. Efficacy and safety of bisphosphonate therapy in children with osteogenesis imperfecta: a systematic review. Horm Res Paediatr. 2015;84(1):26-42.
Morello R. Osteogenesis imperfecta and therapeutics. Matrix Biol. 2018;71-2:294-312.
Constantino CS, Krzak JJ, Fial AV, et al. Effect of bisphosphonates on function and mobility among childrenwith osteogenesis imperfecta: a systematic review. JBMR Plus. 2019;3(10):e10216.
Götherström C, Westgren M, Shaw SW, et al. Pre- and postnatal transplantation of fetal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience. Stem Cells Transl Med. 2014;3(2):255-264.
Guillot PV, Cook HT, Pusey CD, et al. Transplantation of human fetal mesenchymal stem cells improves glomerulopathy in a collagen type I alpha 2-deficient mouse. J Pathol. 2008;214(5):627-636.
Vanleene M, Saldanha Z, Cloyd KL, et al. Transplantation of human fetal blood stem cells in the osteogenesis imperfecta mouse leads to improvement in multiscale tissue properties. Blood. 2011;117(3):1053-1060.
Li F, Wang X, Niyibizi C. Distribution of single-cell expanded marrow derived progenitors in a developing mouse model of osteogenesis imperfecta following systemic transplantation. Stem Cells. 2007;25(12):3183-3193.
Otsuru S, Gordon PL, Shimono K, et al. Transplanted bone marrow mononuclear cells and MSCs impart clinical benefit to children with osteogenesis imperfecta through different mechanisms. Blood. 2012;120(9):1933-1941.
Chamberlain JR, Schwarze U, Wang PR, et al. Gene targeting in stem cells from individuals with osteogenesis imperfecta. Science. 2004;303(5661):1198-1201.
Castelein RM, Hasler C, Helenius I, et al. Complex spine deformities in young patients with severe osteogenesis imperfecta: current concepts review. J Child Orthop. 2019;13(1):22-32.
Kaneto CM, Pereira Lima PS, Prata KL, et al. Gene expression profiling of bone marrow mesenchymal stem cells from osteogenesis imperfecta patients during osteoblast differentiation. Eur J Med Genet. 2017;60(6):326-334.
Pochampally RR, Horwitz EM, DiGirolamo CM, et al. Correction of a mineralization defect by overexpression of a wild-type cDNA for COL1A1 in marrow stromal cells (MSCs) from a patient with osteogenesis imperfecta: a strategy for rescuing mutations that produce dominant-negative protein defects. Gene Ther. 2005;12(14):1119-1125.
Bionaz M, Monaco E, Wheeler MB. Transcription adaptation during in vitro adipogenesis and osteogenesis of porcine mesenchymal stem cells: dynamics of pathways, biological processes, up-stream regulators, and gene networks. PLoS One. 2015;10(9):e0137644.
Chen Q, Shou P, Zheng C, et al. Fate decision of mesenchymal stem cells: adipocytes or osteoblasts? Cell Death Differ. 2016;23(7):1128-1139.
Varelas X. The hippo pathway effectors TAZ and YAP in development, homeostasis and disease. Development. 2014;141(8):1614-1626.
Wang S, Xie F, Chu F, et al. YAP antagonizes innate antiviral immunity and is targeted for lysosomal degradation through IKKɛ-mediated phosphorylation. Nat Immunol. 2017;18(7):733-743.
Zhao B, Tumaneng K, Guan KL. The hippo pathway in organ size control, tissue regeneration and stem cell self-renewal. Nat Cell Biol. 2011;13(8):877-883.
Pan D. Hippo signaling in organ size control. Genes Dev. 2007;21(8):886-897.
Yu FX, Zhao B, Guan KL. Hippo pathway in organ size control, tissue homeostasis, and cancer. Cell. 2015;163(4):811-828.
Pan JX, Xiong L, Zhao K, et al. YAP promotes osteogenesis and suppresses adipogenic differentiation by regulating beta-catenin signaling. Bone Res. 2018;6:18.
Liu Y, Wang J, Liu S, et al. A novel transgenic murine model with persistently brittle bones simulating osteogenesis imperfecta type I. Bone. 2019;127:646-655.
Xiong J, Almeida M, O'Brien CA. The YAP/TAZ transcriptional co-activators have opposing effects at different stages of osteoblast differentiation. Bone. 2018;112:1-9.
Tournis S, Dede AD. Osteogenesis imperfecta-a clinical update. Metabolism. 2018;80:27-37.
Niklason LE. Understanding the extracellular matrix to enhance stem cell-based tissue regeneration. Cell Stem Cell. 2018;22(3):302-305.
Nair AK, Gautieri A, Chang SW, et al. Molecular mechanics of mineralized collagen fibrils in bone. Nat Commun. 2013;4:1724.
Liu X, Long X, Liu W, et al. Type I collagen induces mesenchymal cell differentiation into myofibroblasts through YAP-induced TGF-β1 activation. Biochimie. 2018;150:110-130.
Wittkowske C, Reilly GC, Lacroix D, et al. In vitro bone cell models: impact of fluid shear stress on bone formation. Front Bioeng Biotechnol. 2016;4:87.
Horwitz EM, Prockop DJ, Gordon PL, et al. Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta. Blood. 2001;97(5):1227-1231.
Guillot PV, Abass O, Bassett JH, et al. Intrauterine transplantation of human fetal mesenchymal stem cells from first-trimester blood repairs bone and reduces fractures in osteogenesis imperfecta mice. Blood. 2008;111(3):1717-1725.
Götherström C, Walther-Jallow L. Stem cell therapy as a treatment for osteogenesis imperfecta. Curr Osteoporos Rep. 2020;18(4):337-343.
Solomon LM, Brockman-Lee SA. Embryonic stem cells in science and medicine, part II: law, ethics, and the continuing need for dialogue. Gend Med. 2008;5(1):3-9.
Grompe M. Alternative energy for embryonic stem cell research. Nat Rep Stem Cells. 2007. https://doi.org/10.1038/stemcells.2007.100.
Lindroos B, Suuronen R, Miettinen S. The potential of adipose stem cells in regenerative medicine. Stem Cell Rev Rep. 2011;7(2):269-291.
Si Z, Wang X, Sun C, et al. Adipose-derived stem cells: sources, potency, and implications for regenerative therapies. Biomed Pharmacother. 2019;114:108765.
Niemeyer P, Kornacker M, Mehlhorn A, et al. Comparison of immunological properties of bone marrow stromal cells and adipose tissue-derived stem cells before and after osteogenic differentiation in vitro. Tissue Eng. 2007;13(1):111-121.
Gioia R, Panaroni C, Besio R, et al. Impaired osteoblastogenesis in a murine model of dominant osteogenesis imperfecta: a new target for osteogenesis imperfecta pharmacological therapy. Stem Cells. 2012;30(7):1465-1476.
Silver FH, Landis WJ. Deposition of apatite in mineralizing vertebrate extracellular matrices: a model of possible nucleation sites on type I collagen. Connect Tissue Res. 2011;52(3):242-254.
Dupont S, Morsut L, Aragona M, et al. Role of YAP/TAZ in mechanotransduction. Nature. 2011;474(7350):179-183.
Mo JS. The role of extracellular biophysical cues in modulating the hippo-YAP pathway. BMB Rep. 2017;50(2):71-78.
Komatsu N, Kajiya M, Motoike S, et al. Type I collagen deposition via osteoinduction ameliorates YAP/TAZ activity in 3D floating culture clumps of mesenchymal stem cell/extracellular matrix complexes. Stem Cell Res Ther. 2018;9(1):342.
Byun MR, Kim AR, Hwang JH, et al. FGF2 stimulates osteogenic differentiation through ERK induced TAZ expression. Bone. 2014;58:72-80.
Holloway L, Moynihan S, Abrams SA, et al. Effects of oligofructose-enriched inulin on intestinal absorption of calcium and magnesium and bone turnover markers in postmenopausal women. Br J Nutr. 2007;97(2):365-372.
Tjellesen L, Christiansen C, Rødbro P. Effect of 1,25-dihydroxyvitamin D3 on biochemical indices of bone turnover in postmenopausal women. Acta Med Scand. 1984;215(5):411-415.
Liu PY, Hoey KA, Mielke KL, et al. A randomized placebo-controlled trial of short-term graded transdermal estradiol in healthy gonadotropin-releasing hormone agonist-suppressed pre- and postmenopausal women: effects on serum markers of bone turnover, insulin-like growth factor-I, and osteoclastogenic mediators. J Clin Endocrinol Metab. 2005;90(4):1953-1960.