Programming gene and engineered-cell therapies with synthetic biology

Gene and engineered-cell therapies promise to treat diseases by genetically modifying cells to carry out therapeutic tasks. Although the field has had some success in treating monogenic disorders and hematological malignancies, current approaches are limited to overexpression of one or a few transge...

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Veröffentlicht in:Science (American Association for the Advancement of Science) Jg. 359; H. 6376
Hauptverfasser: Kitada, Tasuku, DiAndreth, Breanna, Teague, Brian, Weiss, Ron
Format: Journal Article
Sprache:Englisch
Veröffentlicht: United States 09.02.2018
Schlagworte:
Cell Engineering - methods
Cell- and Tissue-Based Therapy
Cellular Reprogramming Techniques
DNA - genetics
Gene Expression
Gene Regulatory Networks
Genes, Synthetic
Genetic Engineering - methods
Genetic Therapy
Humans
Recombinant Fusion Proteins
RNA - genetics
Synthetic Biology - methods
Transgenes
ISSN:1095-9203, 1095-9203
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Zusammenfassung:Gene and engineered-cell therapies promise to treat diseases by genetically modifying cells to carry out therapeutic tasks. Although the field has had some success in treating monogenic disorders and hematological malignancies, current approaches are limited to overexpression of one or a few transgenes, constraining the diseases that can be treated with this approach and leading to potential concerns over safety and efficacy. Synthetic gene networks can regulate the dosage, timing, and localization of gene expression and therapeutic activity in response to small molecules and disease biomarkers. Such "programmable" gene and engineered-cell therapies will provide new interventions for incurable or difficult-to-treat diseases.
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ISSN:1095-9203
1095-9203
DOI:10.1126/science.aad1067