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Frontiers of Engineering Reports on Leading-Edge Engineering from the 2019 Symposium (2020) / Chapter Skim
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Empowering Genome Editing Through Standards - Samantha Maragh
Pages 55-62

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From page 55...
... is the entire complement of an organism's genetic code and is housed in the basic functional unit of life, the cell. Genome engineering involves tools and techniques to target a specific sequence in a genome and alter the genetic code (genome editing)
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From page 56...
... . APPLICATIONS OF GENOME EDITING Genome editing is being pursued globally by government, academic, and private sectors to transform medicine and bioscience to enable previously impossible advances in areas such as basic biology research, gene therapy, synthetic biology, novel antimicrobials and antivirals, biomanufacturing, agriculture, and food production (Barrangou and Doudna 2016)
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From page 57...
... Technical Limitations Because of technical limitations on the ability to measure the sequence of individual cells at high throughput, edited sequence confirmation involves a bulk measurement sampling of the genomes from a heterogeneously edited cell pool that may contain both intended and unintended edits even at the target site. In addition, because of biological limitations, particularly for human therapeutics, this heterogeneous pool of cells may be the final product.
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From page 58...
... Even with reliable sequence data in hand, there's still the challenge of interpreting the significance of unintended edits and understanding whether edited cells are fit for the intended purpose. Finally, a genome-edited product must be manufactured.
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From page 59...
... Standards for the safe and efficient manufacture of engineered cell products will likely require a paradigm shift and disruptive technologies to address the particular challenges of manufacturing living cells or manufacturing genome editing delivery systems for in vivo editing at large scale. FIGURE 2  Genome editing process overview in context of opportunities for standardization.
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From page 60...
... . Further progress can be made through the development of a suite of tools and technology employing a multidisciplinary approach to address unmet measurement needs such as single cell editing detection and in vivo tracking and monitoring of edited cells once introduced into the environment or a live organism (e.g., in a human for therapeutic treatment)
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From page 61...
... 2017. In vivo target gene activation via CRISPR/Cas9-mediated trans-epigenetic modulation.
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Key Terms

  • genome engineering
  • genetic code
  • edited cells
  • vivo editing
  • editing process

  • target site
  • analysis pipelines
  • editing delivery
  • sequencing methods
  • sequencing data

  • technical limitations
  • relative abundance
  • technically challenging
  • unintended edits
  • intended purpose

  • plant genome
  • enhanced genome
  • nist genome
  • editing genome
  • manufacturing genome

  • eutic genome
  • efficient genome
  • entire genome
  • herapeutic genome
  • existing genome

  • changed genetic
  • specific sequence
  • editing detection
  • human diseases
  • engineering matures


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