Christ George J, Siriwardane Mevan L, de Coppi Paolo
Wake Forest Institute for Regenerative Medicine Winston-Salem, NC, USA ; Laboratory of Regenerative Therapeutics, Deptartment of Biomedical Engineering and Orthopaedic Surgery, University of Virginia Charlottesville, VA, USA.
Wake Forest Institute for Regenerative Medicine Winston-Salem, NC, USA.
Front Pharmacol. 2015 Apr 10;6:53. doi: 10.3389/fphar.2015.00053. eCollection 2015.
Congenital malformations frequently involve either skeletal, smooth or cardiac tissues. When large parts of those tissues are damaged, the repair of the malformations is challenged by the fact that so much autologous tissue is missing. Current treatments require the use of prostheses or other therapies and are associated with a significant morbidity and mortality. Nonetheless, affected children have generally good survival rates and mostly normal schooling. As such, new therapeutic modalities need to represent significant improvements with clear safety profiles. Regenerative medicine and tissue engineering technologies have the potential to dramatically improve the treatment of any disease or disorder involving a lack of viable tissue. With respect to congenital soft tissue anomalies, the development of, for example, implantable muscle constructs would provide not only the usual desired elasticity and contractile proprieties, but should also be able to grow with the fetus and/or in the postnatal life. Such an approach would eliminate the need for multiple surgeries. However, the more widespread clinical applications of regenerative medicine and tissue engineering technologies require identification of the optimal indications, as well as further elucidation of the precise mechanisms and best methods (cells, scaffolds/biomaterials) for achieving large functional tissue regeneration in those clinical indications. In short, despite some amazing scientific progress, significant safety and efficacy hurdles remain. However, the rapid preclinical advances in the field bode well for future applications. As such, translational researchers and clinicians alike need be informed and prepared to utilize these new techniques for the benefit of their patients, as soon as they are available. To this end, we review herein, the clinical need(s), potential applications, and the relevant preclinical studies that are currently guiding the field toward novel therapeutics.
先天性畸形常常累及骨骼、平滑肌或心脏组织。当这些组织的大部分受损时，由于大量自体组织缺失，畸形修复面临挑战。目前的治疗方法需要使用假体或其他疗法，且伴有较高的发病率和死亡率。尽管如此，患病儿童的总体存活率较高，大多能正常上学。因此，新的治疗方式需要有显著改进且安全性明确。再生医学和组织工程技术有潜力显著改善任何涉及缺乏存活组织的疾病或病症的治疗。就先天性软组织异常而言，例如可植入肌肉构建体的开发不仅能提供通常所需的弹性和收缩特性，还应能够随着胎儿生长和/或在出生后生长。这种方法将消除多次手术的必要性。然而，再生医学和组织工程技术更广泛的临床应用需要确定最佳适应症，以及进一步阐明在这些临床适应症中实现大型功能性组织再生的精确机制和最佳方法（细胞、支架/生物材料）。简而言之，尽管取得了一些惊人的科学进展，但仍存在重大的安全性和有效性障碍。然而，该领域临床前的快速进展预示着未来应用前景良好。因此，转化研究人员和临床医生都需要了解并准备好在这些新技术可用时，为患者的利益加以利用。为此，我们在此回顾临床需求、潜在应用以及目前指导该领域走向新型疗法的相关临床前研究。
