Current Genetic Medicine Reports

, Volume 5, Issue 1, pp 35–43 | Cite as

Editing the Genome: Prospects, Progress, Implications, and Cautions

  • Nancy M. P. KingEmail author
  • Pat C. Lord
  • Douglas E. Lemley
Ethics in Genetic Medicine (L Parker, Section Editor)
Part of the following topical collections:
  1. Ethics in Genetic Medicine


Purpose of Review

Clinicians, clinical researchers, and the general public all need to consider the implications of rapid advances in gene editing technology, which have given rise not only to new hope for the development of corrective interventions for genetic defects and many other diseases and conditions but also to many ethical, social, and policy concerns.

Recent Findings

Gene editing tools are potentially both more precise and more accurate than current gene transfer interventions; in addition, the discovery and development of the CRISPR/Cas9 system has made gene editing research considerably easier, faster, and cheaper. As a result, long-standing debates about the safety, efficacy, affordability, ethical and social acceptability, oversight, and control of efforts to pursue human germline alteration and enhancement have been rekindled. Added to these debates are controversies about newly discovered capacities to use gene editing to reshape the environment by altering or eliminating non-human species (insects in particular) that pose threats to humans.


This review examines the science of the new gene editing boom in its social and historical context and discusses both past and current policy debates and future prospects for ethical consensus on whether, where, when, and how to move forward with clinical research and medical applications.


CRISPR CRISPR/Cas9 ELSI Gene editing Germline gene editing Genetic enhancement 


Compliance with Ethical Standards

Conflict of Interest

Nancy M. P. King, Pat C. Lord, and Douglas E. Lemley declare no conflicts of interest.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.


Papers of particular interest, published recently, have been highlighted as: • Of importance

  1. 1.
    • Hampton T. Ethical and societal questions loom large as gene editing moves closer to the clinic. JAMA. 2016;315(6):546–8. A brief overview of historical and recent scientific developments and ethical and policy debates about genetic intervention research in general and gene editing in particular.CrossRefPubMedGoogle Scholar
  2. 2.
    Coutts MC. Human gene therapy. Kennedy Inst Ethics J. 1994;4(1):63–83.CrossRefPubMedGoogle Scholar
  3. 3.
    Weiss R, Nelson D. Methods faulted in gene test death, teen too ill for therapy, probe finds. Washington Post. 1999;8:A1.Google Scholar
  4. 4.
    Gelsinger P. Jesse’s intent. Guinea pig zero: a journal for human research subjects. 2000. Issue #8. Accessed 12 Oct 2016.
  5. 5.
    Pearson S, Jia H, Kandachi K. China approves first gene therapy. Nature Biotechnol. 2004;22(1):3–4.CrossRefGoogle Scholar
  6. 6.
    Moran N. First gene therapy glybera (finally) gets EMA approval. BioWorld, Thompson Reuters. 2012. Accessed 12 Oct 2016.
  7. 7.
    Ward A. GSK to allow staggered payments for EMA-approved gene therapy. The financial times. 2016. Accessed 12 Oct 2016.
  8. 8.
    Pollack A. Eye treatment closes in on being first gene therapy approved in U.S. New York Times. 2015. p. B3. Accessed 12 Oct 2016.
  9. 9.
    Van den Driessche T, Chuah MK. CRISPR/Cas9 flexes its muscles: in vivo somatic gene editing for muscular dystrophy. Mol Ther. 2016;24:414–6.CrossRefGoogle Scholar
  10. 10.
    Durai S, Mani M, Kandavelou K, Wu J, Porteus MH, Chandrasegaran S. Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells. Nucleic Acids Res. 2005;33(18):5978–90.CrossRefPubMedPubMedCentralGoogle Scholar
  11. 11.
    Boch J. TALEs of genome targeting. Nature Biotechnol. 2011;29(2):135–6.CrossRefGoogle Scholar
  12. 12.
    GeMCRIS website. Accessed 12 Oct 2016.
  13. 13.
    Pollack A. A cell therapy untested in humans saves a baby with cancer. New York Times. 2015;6:B3.Google Scholar
  14. 14.
    Stoddard BL, Fox K. Editorial: CRISPR in nucleic acids research. Nucleic Acids Res. 2016;44:4489–90.CrossRefGoogle Scholar
  15. 15.
    Keener AB. Gene editing: from roots to riches. Scientist. 2016. Accessed 12 Oct 2016.
  16. 16.
    Comfort N. Can we cure genetic diseases without slipping into eugenics? The nation. 2015. Accessed 12 Oct 2016.
  17. 17.
    Specter M. The gene hackers. New Yorker. 2015;16:52–61.Google Scholar
  18. 18.
    Park A. Life: the remix. Time. 2016;4:43–8.Google Scholar
  19. 19.
    Center for Genomics and Society. About human germline gene editing. Accessed 12 Oct 2016.
  20. 20.
    Office of Science Policy, NIH. Next steps on research using animal embryos containing human cells, Under the Poliscope. 2016. Accessed 12 Oct 2016.
  21. 21.
    Regalado A. Top US intelligence official calls gene editing a WMD threat. MIT Technology Rev. 2016. Accessed 12 Oct 2016.
  22. 22.
    Marchant GE, Wallach W. Coordinating technology governance. Issues Sci Technol. 2015;31(4). Accessed 12 Oct 2016.
  23. 23.
    McEwen JE, Boyer JT, Sun KY, et al. The ethical, legal, and social implications program of the National Human Genome Research Institute: reflections on an ongoing experiment. Ann Rev Genomics Hum Genetics. 2014;15:481–505.CrossRefGoogle Scholar
  24. 24.
    Pinker S. The moral imperative for bioethics. Boston Globe. 2015. Accessed 12 Oct 2016.
  25. 25.
    Hall SS. Will we control our genetic destinies? Sci Am. 2016;315:54–61.CrossRefPubMedGoogle Scholar
  26. 26.
    Regalado A. Engineering the perfect baby. MIT Technol Rev. 2015;118(3):27–33.Google Scholar
  27. 27.
    Rogers M. The Pandora’s box Congress, Rolling Stone. 1975.Google Scholar
  28. 28.
    Berg P. Asilomar 1975: DNA modification secured. Nature. 2008;455:290–1.CrossRefPubMedGoogle Scholar
  29. 29.
    Hurlbut JB. Limits of responsibility: genome editing, Asilomar, and the politics of deliberation. Hastings Center Rep. 2015;45(5):11–4.CrossRefGoogle Scholar
  30. 30.
    Hayden EC. Tomorrow’s children. Nature. 2016;530:402–5.CrossRefGoogle Scholar
  31. 31.
    Regalado A. Patients favor changing the genes of the next generation with CRISPR, MIT Technol Rev. 2015. Accessed 12 Oct 2016.
  32. 32.
    National Academies of Sciences, Engineering, and Medicine. International summit on human gene editing: a global discussion–commissioned papers, 2015. Accessed 12 Oct 2016.
  33. 33.
    Sarewitz D. CRISPR: science can’t solve it. Nature. 2015;522:413–4.CrossRefPubMedGoogle Scholar
  34. 34.
    • Lander ES. Brave new genome. NEJM. 2015;373:5–8. Short comprehensive review of ethical and policy issues in gene editing.CrossRefPubMedGoogle Scholar
  35. 35.
    Lander ES. The heroes of CRISPR. Cell. 2016;164(1):18–28.CrossRefPubMedGoogle Scholar
  36. 36.
    Ishino Y, Shinagawa H, Makino K, et al. Nucleotide sequence of the IAP gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product. J Bacteriol. 1987;169(12):5429–33.CrossRefPubMedPubMedCentralGoogle Scholar
  37. 37.
    Mojica FJM, Diez-Villasenor C, Garcia-Martinez J, et al. Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements. J Mol Evol. 2005;60(2):174–82.CrossRefPubMedGoogle Scholar
  38. 38.
    Sternberg SH, Doudna J. Expanding the biologist’s toolkit with CRISPR-Cas9. Mol Cell. 2015;58:568–74.CrossRefPubMedGoogle Scholar
  39. 39.
    • Maeder ML, Gersbach CA. Genome-editing technologies for gene and cell therapy. Mol Biol. 2016;24:430–46. Definitive scientific overview.Google Scholar
  40. 40.
    Jinek M, Chylinski K, Fonfara I, et al. A programmable dual-RNA-guided endonuclease in adaptive bacterial immunity. Science. 2012;337:816–21.CrossRefPubMedGoogle Scholar
  41. 41.
    Gasiunas G, Barrangou R, Horvath P, et al. Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria. Proc Natl Acad Sci U S A. 2012;109(39):15539–40.CrossRefGoogle Scholar
  42. 42.
    Cong L, Ran FA, Cox D, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339:819–23.CrossRefPubMedPubMedCentralGoogle Scholar
  43. 43.
    Doudna JA, Charpentier E. The new frontier of genome engineering with CRISPR-Cas9. Science. 2014;346:1077–86.CrossRefGoogle Scholar
  44. 44.
    Jo YI, Suresh B, Kim H, et al. CRISPR/Cas9 system as an innovative genetic engineering tool: enhancements in sequence specificity and delivery methods. Biochim Biophys Acta. 1856;2015:234–43.Google Scholar
  45. 45.
    Mei Y, Wang Y, Chen H, et al. Recent progress in CRISPR/Cas9 technology. J Genetics Genomics. 2016;43:63–75.CrossRefGoogle Scholar
  46. 46.
    • Baltimore D, Berg P, Botchan M, et al. A prudent path forward for genomic engineering and germline gene modification. Science. 2015;348:36–8. The first (and arguably most influential) group policy recommendations on gene editing.CrossRefPubMedPubMedCentralGoogle Scholar
  47. 47.
    International Society for Stem Cell Research. The ISSCR statement on human germline genome modification. 2015. Accessed 12 Oct 2016.
  48. 48.
    Lanphier E, Urnov F, Haecker SE, et al. Don’t edit the human germ line. Nature. 2015;519:410–1.CrossRefPubMedGoogle Scholar
  49. 49.
    • Liang P, Xu Y, Zhang X, et al. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell. 2015;6:363–72. The first publication on human embryo research using CRISPR.CrossRefPubMedPubMedCentralGoogle Scholar
  50. 50.
    National Institutes of Health. Statement on NIH funding of research using gene-editing technologies in human embryos. 2015. Accessed 12 Oct 2016.
  51. 51.
    Friedmann T, Jonlin EC, King NMP, et al. ASGCT and JSGT joint position statement on human genomic editing. Mol Ther. 2015;23:1282.CrossRefPubMedPubMedCentralGoogle Scholar
  52. 52.
    Hinxton Group. Statement on genome editing technologies and human germline genetic modification. 2015. Accessed 12 Oct 2016.
  53. 53.
    International Bioethics Committee. Report of the IBC on updating its reflection on the human genome and human rights. (Paris: UNESCO). 2015. Accessed 12 Oct 2016.
  54. 54.
    National Institutes of Health. Notice Number NOT-OD-15-158, NIH research involving introduction of human pluripotent cells into non-human vertebrate animal pre-gastrulation embryos. 2015. Accessed 12 Oct 2016.
  55. 55.
    Maynard A. What do you think about scientists creating human-nonhuman hybrids? The National Institutes of Health wants to know. Slate/Future Tense. 2016. Accessed 12 Oct 2016.
  56. 56.
    National Academies of Sciences, Engineering, and Medicine. On human gene editing: International Summit statement. 2015. Accessed 12 Oct 2016.
  57. 57.
    National Academies of Sciences, Engineering, and Medicine. International summit on human gene editing: a global discussion. Washington, DC: National Academies Press; 2015 . Accessed 12 Oct 2016Google Scholar
  58. 58.
    Reardon S. Global summit reveals divergent views on human gene editing. Nature. 2015;528:173.CrossRefPubMedGoogle Scholar
  59. 59.
    National Academies of Sciences, Engineering, and Medicine. Human gene-editing initiative consensus study. Human gene editing: scientific, medical, and ethical considerations. Accessed 12 Oct 2016.
  60. 60.
    Garde D. The cure for ‘bubble boy’ disease will cost $665,000. STAT News. 2016. Accessed 12 Oct 2016.
  61. 61.
    King NMP. Accident and desire. Hastings Center Rep. 2003;33(2):23–30.CrossRefGoogle Scholar
  62. 62.
    Liu KI, Bin Ramli MN, Woo CWA, et al. A chemical-inducible CRISPR-Cas9 system for rapid control of gene editing. Nature Chem Biol. 2016; doi: 10.1038/nchembio.2179.Google Scholar
  63. 63.
    Begley S. They’re going to CRISPR people. What could possibly go wrong? STAT. 2016. Accessed 12 Oct 2016.
  64. 64.
    Belkin L. The made-to-order savior: producing a perfect baby sibling. New York Times Magazine. 2001. Accessed 12 Oct 2016.
  65. 65.
    Callaway E. UK scientists gain licence to edit genes in human embryos. Nature. 2016; doi: 10.1038/nature.2016.19270.PubMedCentralGoogle Scholar
  66. 66.
    Callaway E. Embryo-editing research gathers momentum. Nature. 2016;532:289–90.CrossRefPubMedGoogle Scholar
  67. 67.
    Stein R. Breaking taboo, Swedish scientist seeks to edit DNA of healthy human embryos, Shots Health News from NPR. 2016. Accessed 12 Oct 2016.
  68. 68.
    Flotte R. Therapeutic germ line alteration: has CRISPR/Cas9 technology forced the question? Mol Ther. 2015;26:1–2.Google Scholar
  69. 69.
    Hildt E. Human germline interventions—think first. Frontiers Genetics. 2016; doi: 10.3389/fgene.2016.00081.Google Scholar
  70. 70.
    Sugarman J. Ethics and germline gene editing. EMBO Rep. 2015;16:879–80.CrossRefPubMedPubMedCentralGoogle Scholar
  71. 71.
    Heidari R, Shaw DM, Elger BS. CRISPR and the rebirth of synthetic biology. Sci Eng Ethics. 2016; doi: 10.1007/s11948-016-9768-z.PubMedGoogle Scholar
  72. 72.
    Porteus MH, Dann CT. Genome editing of the germline: broadening the discussion. Mol Ther. 2015;23:980–2.CrossRefPubMedPubMedCentralGoogle Scholar
  73. 73.
    Carroll D. A perspective on the state of genome editing. Mol Ther. 2016;24:412–3.CrossRefPubMedPubMedCentralGoogle Scholar
  74. 74.
    Zhai X, Ng V, Lie R. No ethical divide between China and the West in human embyro research. Dev World Bioeth. 2016;16:116–20.CrossRefPubMedGoogle Scholar
  75. 75.
    Savulescu J, Pugh J, Douglas T, Gyngell C. The moral imperative to continue gene editing research on human embryos. Protein Cell. 2015;6:476–9.CrossRefPubMedPubMedCentralGoogle Scholar
  76. 76.
    Gallagher J. Embryo engineering a moral duty, says top scientist. BBC News. 2015. Accessed 12 Oct 2016.
  77. 77.
    • Chapman AR, Frankel MS. Human inheritable genetic modification: assessing scientific, ethical, religious, and policy issues. Washington DC: American Association for the Advancement of Science; 2000 . Accessed 12 Oct 2016. Definitive examination.Google Scholar
  78. 78.
    Walters L, Palmer JG. The ethics of human gene therapy. New York: Oxford University Press; 1997.Google Scholar
  79. 79.
    Juengst ET. Can enhancement be distinguished from prevention in genetic medicine? J Med Philos. 1997;22:125–42.CrossRefPubMedGoogle Scholar
  80. 80.
    • Juengst ET, Moseley D. Human enhancement. In: Stanford encyclopedia of philosophy. ). Accessed 12 Oct 2016. Clear and detailed examination .
  81. 81.
    Shaw D, Dondorp W, Geijsen N, de Wert G. Creating human organs in chimaera pigs: an ethical source of immunocompatible organs? J Med Ethics. 2015;41:970–4.CrossRefPubMedGoogle Scholar
  82. 82.
    Walsh F. US bid to grow human organs for transplant inside pigs. BBC News. 2016. Accessed 12 Oct 2016.
  83. 83.
    • National Academies of Sciences, Engineering, and Medicine. Gene drives on the horizon. 2016. Accessed 12 Oct 2016. Comprehensive report on the science and the policy implications of gene drives .
  84. 84.
    Begley S. Monsanto licenses CRISPR technology to modify crops—with key restrictions. STAT/Scientific American. 2016. Accessed 12 Oct 2016.
  85. 85.
    Goldman J. Harnessing the power of gene drives to save wildlife. Sci Am. 2016. Accessed 12 Oct 2016.
  86. 86.
    Swetlitz I. ‘Gene drive’ organisms should be tested in field trials, not widely released, experts say. STAT. 2016. Accessed 12 Oct 2016.

Copyright information

© Springer Science + Business Media New York 2017

Authors and Affiliations

  • Nancy M. P. King
    • 1
    Email author
  • Pat C. Lord
    • 2
  • Douglas E. Lemley
    • 3
  1. 1.Department of Social Sciences and Health PolicyWake Forest School of MedicineWinston-SalemUSA
  2. 2.Department of BiologyWake Forest UniversityWinston-SalemUSA
  3. 3.Graduate Program in BioethicsWake Forest UniversityWinston-SalemUSA

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