期刊
NATURE REVIEWS RHEUMATOLOGY
卷 11, 期 4, 页码 234-242出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nrrheum.2015.28
关键词
-
类别
资金
- AO Foundation
- NIH from National Institute for Arthritis and Musculoskeletal Skin Diseases [R01 AR050243, R01 AR052809, R01 AR43623, R21 AR049606, R01 AR048566, R01 AR057422, R01 AR051085]
- National Institute on Aging [1P01AG043376-01A1]
- National Center for Advancing Translation Sciences [X01 NS066865]
- Department of Defense (the Armed Forces Institute of Regenerative Medicine II) [W81XWH-13-2-0052]
Injuries to the musculoskeletal system are common, debilitating and expensive. In many cases, healing is imperfect, which leads to chronic impairment. Gene transfer might improve repair and regeneration at sites of injury by enabling the local, sustained and potentially regulated expression of therapeutic gene products; such products include morphogens, growth factors and anti-inflammatory agents. Proteins produced endogenously as a result of gene transfer are nascent molecules that have undergone post-translational modification. In addition, gene transfer offers particular advantages for the delivery of products with an intracellular site of action, such as transcription factors and noncoding RNAs, and proteins that need to be inserted into a cell compartment, such as a membrane. Transgenes can be delivered by viral or nonviral vectors via in vivo or ex vivo protocols using progenitor or differentiated cells. The first gene transfer clinical trials for osteoarthritis and cartilage repair have already been completed. Various bone-healing protocols are at an advanced stage of development, including studies with large animals that could lead to human trials. Other applications in the repair and regeneration of skeletal muscle, intervertebral disc, meniscus, ligament and tendon are in preclinical development. In addition to scientific, medical and safety considerations, clinical translation is constrained by social, financial and logistical issues.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据