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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Published in:	Science (American Association for the Advancement of Science) Vol. 359; no. 6376
Main Authors:	Kitada, Tasuku, DiAndreth, Breanna, Teague, Brian, Weiss, Ron
Format:	Journal Article
Language:	English
Published:	United States 09.02.2018
Subjects:	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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Abstract	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.
AbstractList	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. 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.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.
Author	Teague, Brian Weiss, Ron Kitada, Tasuku DiAndreth, Breanna
Author_xml	– sequence: 1 givenname: Tasuku orcidid: 0000-0003-2265-3363 surname: Kitada fullname: Kitada, Tasuku organization: Synthetic Biology Center, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA – sequence: 2 givenname: Breanna orcidid: 0000-0002-0302-6834 surname: DiAndreth fullname: DiAndreth, Breanna organization: Synthetic Biology Center, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA – sequence: 3 givenname: Brian orcidid: 0000-0002-9833-2817 surname: Teague fullname: Teague, Brian organization: Synthetic Biology Center, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA – sequence: 4 givenname: Ron orcidid: 0000-0003-0396-2443 surname: Weiss fullname: Weiss, Ron email: rweiss@mit.edu organization: Synthetic Biology Center, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA. rweiss@mit.edu
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Copyright	Copyright © 2018 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.
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Title	Programming gene and engineered-cell therapies with synthetic biology
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