Pelinkovic D, Martinek V, Engelhardt M, Lee J Y, Fu F, Huard J
Department of Orthopaedic Surgery and Molecular Genetics and Biochemistry, Children's Hospital of Pittsburgh, USA.
Z Orthop Ihre Grenzgeb. 2000 Sep-Oct;138(5):402-6. doi: 10.1055/s-2000-10168.
Muscle-based somatic gene therapy is a novel way to alleviate a biochemical deficiency.
Muscle-derived cells are very promising in the field of gene therapy and tissue engineering. First, most muscle tissue is accessible by injection. Second, muscle tissue consists of multinucleated, postmitotic myofibers, which enable a long-term expression of the transduced gene. Third, muscle tissue can be biopsied easily. It is available in abundance and the biopsy does not compromise the health and function of the patient. Finally, muscle tissue is highly vascularized, which makes systemic delivery feasible.
Muscle-derived cells can promote muscle healing and bone healing. Implanted cells maintain a long-term transgene expression of therapeutic proteins. Isolated, muscle-derived stem cells can differentiate in osteoblasts.
Based on these characteristics, we present four possible applications: inherited muscular diseases, muscle injury, bone healing, and intraarticular disorders.
基于肌肉的体细胞基因治疗是缓解生化缺陷的一种新方法。
肌肉来源的细胞在基因治疗和组织工程领域非常有前景。首先，大多数肌肉组织可通过注射进入。其次，肌肉组织由多核、有丝分裂后肌纤维组成，这使得转导基因能够长期表达。第三，肌肉组织易于活检。其来源丰富，活检不会损害患者的健康和功能。最后，肌肉组织血管高度丰富，这使得全身给药可行。
肌肉来源的细胞可促进肌肉愈合和骨愈合。植入的细胞能维持治疗性蛋白质的长期转基因表达。分离出的肌肉来源干细胞可分化为成骨细胞。
基于这些特性，我们提出了四种可能的应用：遗传性肌肉疾病、肌肉损伤、骨愈合和关节内疾病。
