Gene therapy approaches to regenerating the musculoskeletal system
نویسندگان
چکیده
منابع مشابه
Current concepts in gene therapy of the musculoskeletal system.
The purpose of this article is to review the remarkable progress in the field of musculoskeletal system gene therapy. Since the introduction of this concept 15 years much of the preclinical and clinical data have emerged. The original target, rheumatoid arthritis, has been subjected to clinical phase II efficacy protocol, and osteoarthritis gene therapy efficacy is being thoroughly investigated...
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There have been rapid advances in the development of non-viral techniques for gene transfer. Although viruses are highly evolved to infect mammalian cells, they have several limitations. The general theme is to mimic the advantageous components of viral systems whilst separating them from their limiting functions. The systems described here are broadly divided into true non-viral techniques and...
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Hemoglobinopathies such as thalassemia and sickle cell anemia are potentially amenable to gene therapy. Applicable gene therapy strategies can be divided into four categories: those that replace the faulty gene with a complete transcript ional unit , those that activate transcription of fetal hemoglobin genes, those that modify the endogenous gene itself , and those that attempt to repair the d...
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The aim of the present study was to evaluate the efficacy of novel nonviral gene delivery systems in cells of musculoskeletal origin. Primary cultures of lapine skeletal muscle cells, lapine articular chondrocytes, human cells from fibrous dysplasia and cell lines established from human osteosarcoma (SAOS-2), chondrosarcoma (CS-1), murine skeletal myoblasts (L8) and fibroblasts (NIH 3T3) were t...
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Where we started In the present era of the nearing completion of the nucleotide sequences of the human genome and of several model organisms, it is easy to overlook the common viewpoint of 20 years ago, especially among neuroscientists. It was clear at that time intellectually that the genome encoded the protein set and that the proteins provided the hardware for the biochemical operation of th...
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ژورنال
عنوان ژورنال: Nature Reviews Rheumatology
سال: 2015
ISSN: 1759-4790,1759-4804
DOI: 10.1038/nrrheum.2015.28