Title |
Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies
|
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Published in |
Molecular and Cellular Pediatrics, November 2015
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DOI | 10.1186/s40348-015-0022-6 |
Pubmed ID | |
Authors |
Justin S. Antony, Alexander Dewerth, Ashiqul Haque, Rupert Handgretinger, Michael S.D. Kormann |
Abstract |
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. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 2 | 8% |
Canada | 2 | 8% |
Australia | 2 | 8% |
France | 1 | 4% |
Malaysia | 1 | 4% |
Spain | 1 | 4% |
Unknown | 15 | 63% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 22 | 92% |
Scientists | 1 | 4% |
Science communicators (journalists, bloggers, editors) | 1 | 4% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Turkey | 1 | 2% |
United States | 1 | 2% |
Unknown | 45 | 96% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 9 | 19% |
Researcher | 8 | 17% |
Student > Ph. D. Student | 6 | 13% |
Student > Bachelor | 5 | 11% |
Student > Doctoral Student | 4 | 9% |
Other | 8 | 17% |
Unknown | 7 | 15% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 12 | 26% |
Medicine and Dentistry | 9 | 19% |
Agricultural and Biological Sciences | 9 | 19% |
Immunology and Microbiology | 2 | 4% |
Engineering | 2 | 4% |
Other | 5 | 11% |
Unknown | 8 | 17% |