Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies.
Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies.
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DOI：
10.1186/s40348-015-0022-6
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发表时间：
2015-12
期刊：
影响因子：
--
通讯作者：
Kormann MS
中科院分区：
文献类型：
--
作者：
Antony JS;Dewerth A;Haque A;Handgretinger R;Kormann MS
The immunogenicity and limited stability of conventional messenger RNA (mRNA) has traditionally restricted its potential therapeutic use. In 1992, the first clinical application of mRNA was reported as a potential protein-replacement therapy; however, subsequent investigations have not been made for almost two decades. Recent developments, including increased stability, controlling immunogenicity, as well as utilization of mRNA encoding zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR-Cas9, have implicated modified mRNA as a very promising option for cancer immunotherapy, vaccines, protein expression replacement, and genome editing. This review aims to offer a summary of our present understanding of and improvements in mRNA-based drug technologies, along with a focus on the role in therapeutic options for pediatric respiratory diseases and hemoglobinopathies. This mini review summarizes the recent advances in modified mRNA-based therapy and its potential therapeutic effect in treating major pediatric diseases.
