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| Content Provider | World Health Organization (WHO)-Global Index Medicus |
|---|---|
| Author | Yin, Hao Kanasty, Rosemary L. Eltoukhy, Ahmed A. Vegas, Arturo J. Dorkin, J. Robert Anderson, Daniel G. |
| Description | Country affiliation: United States Author Affiliation: Yin H ( David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA.); Kanasty RL ( 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Chemical Engineering, MIT, Cambridge, Massachusetts 02142, USA.); Eltoukhy AA ( David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA.); Vegas AJ ( 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Anesthesiology, Children's Hospital Boston, Boston, Massachusetts 02115, USA.); Dorkin JR ( 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Biology, MIT, Cambridge, Massachusetts 02142, USA.); Anderson DG ( 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Chemical Engineering, MIT, Cambridge, Massachusetts 02142, USA. [3] Department of Anesthesiology, Children's Hospital Boston, Boston, Massac) |
| Abstract | Gene-based therapy is the intentional modulation of gene expression in specific cells to treat pathological conditions. This modulation is accomplished by introducing exogenous nucleic acids such as DNA, mRNA, small interfering RNA (siRNA), microRNA (miRNA) or antisense oligonucleotides. Given the large size and the negative charge of these macromolecules, their delivery is typically mediated by carriers or vectors. In this Review, we introduce the biological barriers to gene delivery in vivo and discuss recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems, some of which are currently undergoing testing in clinical trials. The diversity of these systems highlights the recent progress of gene-based therapy using non-viral approaches. |
| File Format | HTM / HTML |
| ISSN | 14710056 |
| Issue Number | 8 |
| Volume Number | 15 |
| e-ISSN | 14710064 |
| Journal | Nature Reviews Genetics |
| Language | English |
| Publisher | Nature Publishing Group |
| Publisher Date | 2014-08-01 |
| Publisher Place | Great Britain (UK) |
| Access Restriction | One Nation One Subscription (ONOS) |
| Subject Keyword | Discipline Genetics Genetic Therapy Genetic Vectors Genetics Animals Dna Gene Transfer Techniques Administration & Dosage Humans Rna Journal Article Research Support, N.i.h., Extramural Research Support, Non-u.s. Gov't Research Support, U.s. Gov't, Non-p.h.s. Review |
| Content Type | Text |
| Resource Type | Article |
| Subject | Genetics Molecular Biology Genetics (clinical) |
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