成肌细胞移植：一种可能的严重儿科疾病的手术治疗方法。
Myoblast transplantation: a possible surgical treatment for a severe pediatric disease.
机构信息
Department of General Surgery and Surgical Specialties, University of Modena and Reggio Emilia Medical School, Surgical Clinic, 41100 Modena, Italy.
出版信息
Surg Today. 2010 Oct;40(10):902-8. doi: 10.1007/s00595-009-4242-z. Epub 2010 Sep 25.
Abstract
Duchenne muscular dystrophy (DMD) is a genetic X-linked recessive orphan disease that affects approximately 1 in 3 500 male births. Boys with DMD have progressive and predictable muscle destruction due to the absence of dystrophin, a protein present under the muscle fiber membrane. This absence induces contraction-related membrane damage and activation of inflammatory necrosis and fibrosis, leading to cardiac/diaphragmatic failure and death. The authors support the therapeutic role of myoblast transplantation in DMD, and describe the history and rationale for such an approach.
摘要
杜氏肌营养不良症（DMD）是一种遗传性 X 连锁隐性罕见疾病，影响约每 3500 名男婴中的 1 名。患有 DMD 的男孩由于缺乏肌营养不良蛋白，导致肌肉纤维膜下的蛋白质缺失，从而出现进行性和可预测的肌肉破坏。这种缺失会导致与收缩相关的膜损伤以及炎症性坏死和纤维化的激活，导致心脏/膈肌衰竭和死亡。作者支持肌母细胞移植在 DMD 中的治疗作用，并描述了这种方法的历史和原理。
相似文献
1
Myoblast transplantation: a possible surgical treatment for a severe pediatric disease.
Surg Today. 2010 Oct;40(10):902-8. doi: 10.1007/s00595-009-4242-z. Epub 2010 Sep 25.
2
Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy.
Pediatr Transplant. 2010 Nov;14(7):813-9. doi: 10.1111/j.1399-3046.2010.01377.x.
3
[Duchenne muscular dystrophy: rational basis, state of the art].
Recenti Prog Med. 2006 Sep;97(9):441-7.
4
Induction of CCAAT/Enhancer-Binding Protein β Expression With the Phosphodiesterase Inhibitor Isobutylmethylxanthine Improves Myoblast Engraftment Into Dystrophic Muscle.
Stem Cells Transl Med. 2016 Apr;5(4):500-10. doi: 10.5966/sctm.2015-0169. Epub 2016 Mar 3.
5
Hematopoietic cell transplantation provides an immune-tolerant platform for myoblast transplantation in dystrophic dogs.
Mol Ther. 2008 Jul;16(7):1340-6. doi: 10.1038/mt.2008.102. Epub 2008 May 27.
6
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update.
Genes (Basel). 2020 Jul 23;11(8):837. doi: 10.3390/genes11080837.
7
[Co-transplantation of myoblasts and schwann cells in the therapy of Duchenne muscular dystrophy].
Sichuan Da Xue Xue Bao Yi Xue Ban. 2011 Jan;42(1):101-5.
8
Effects of omega-3 on matrix metalloproteinase-9, myoblast transplantation and satellite cell activation in dystrophin-deficient muscle fibers.
Cell Tissue Res. 2017 Sep;369(3):591-602. doi: 10.1007/s00441-017-2640-x. Epub 2017 Jun 17.
9
Assessment of a symptomatic Duchenne muscular dystrophy carrier 20 years after myoblast transplantation from her asymptomatic identical twin sister.
Neuromuscul Disord. 2013 Jul;23(7):575-9. doi: 10.1016/j.nmd.2013.04.007. Epub 2013 May 31.
10
Cardiac Protection after Systemic Transplant of Dystrophin Expressing Chimeric (DEC) Cells to the mdx Mouse Model of Duchenne Muscular Dystrophy.
Stem Cell Rev Rep. 2019 Dec;15(6):827-841. doi: 10.1007/s12015-019-09916-0.
引用本文的文献
1
Genome engineering: a new approach to gene therapy for neuromuscular disorders.
Nat Rev Neurol. 2017 Nov;13(11):647-661. doi: 10.1038/nrneurol.2017.126. Epub 2017 Sep 29.
2
Generation of skeletal muscle cells from embryonic and induced pluripotent stem cells as an in vitro model and for therapy of muscular dystrophies.
J Cell Mol Med. 2012 Jul;16(7):1353-64. doi: 10.1111/j.1582-4934.2011.01498.x.
本文引用的文献
1
Central tolerance to myogenic cell transplants does not include muscle neoantigens.
Transplantation. 2008 Jun 27;85(12):1791-801. doi: 10.1097/TP.0b013e31817726bc.
2
Ischemic central necrosis in pockets of transplanted myoblasts in nonhuman primates: implications for cell-transplantation strategies.
Transplantation. 2007 Nov 27;84(10):1307-15. doi: 10.1097/01.tp.0000288322.94252.22.
3
Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.
Biochem Soc Trans. 2007 Aug;35(Pt 4):826-8. doi: 10.1042/BST0350826.
4
Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries.
BMC Mol Biol. 2007 Jul 2;8:57. doi: 10.1186/1471-2199-8-57.
5
Human muscle precursor cells give rise to functional satellite cells in vivo.
Neuromuscul Disord. 2007 Aug;17(8):631-8. doi: 10.1016/j.nmd.2007.04.009. Epub 2007 Jun 27.
6
Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse.
Mol Ther. 2007 Sep;15(9):1587-92. doi: 10.1038/sj.mt.6300245. Epub 2007 Jun 19.
7
Calcineurin inhibitors in pediatric renal transplant recipients.
Paediatr Drugs. 2007;9(3):165-74. doi: 10.2165/00148581-200709030-00005.
8
Stem and progenitor cells in skeletal muscle development, maintenance, and therapy.
Mol Ther. 2007 May;15(5):867-77. doi: 10.1038/mt.sj.6300145. Epub 2007 Mar 27.
9
Induction of tolerance across fully mismatched barriers by a nonmyeloablative treatment excluding antibodies or irradiation use.
Cell Transplant. 2006;15(8-9):835-46. doi: 10.3727/000000006783981521.
10
Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy.
Hum Gene Ther. 2007 Jan;18(1):18-26. doi: 10.1089/hum.2006.093.
Zotero 插件