Reactions to the National Academies/Royal Society Report on<i>Heritable Human Genome Editing</i>

Angrist, Misha; Barrangou, Rodolphe; Βaylis, Françoise; Brokowski, Carolyn; Burgio, Gaétan; Caplan, Arthur L.; Chapman, C. P.; Church, George M.; Cook-Deegan, Robert; Cwik, Bryan; Doudna, Jennifer A.; Evans, John H.; Greely, Henry T.; Hercher, Laura; Hurlbut, J. Benjamin; Hynes, Richard O.; Ishii, Tetsuya; Kiani, Samira; Lee, Latasha Hoskins; Levrier, Guillaume; Liu, David R.; Lunshof, Jeantine E.; Macintosh, Kerry Lynn; Mathews, Debra J. H.; Meslin, Eric M.; Mills, Peter H.R.; Montoliu, Lluı́s; Musunuru, Kiran; Nicol, Dianne; O’Neill, Helen C.; Qiu, Ren-Zong; Ranisch, Robert; Sherkow, Jacob S.; Soni, Sheetal; Terry, Sharon F.; Topol, Eric J.; Williamson, Robert; Zhang, Feng; Davies, Kevin

doi:10.1089/crispr.2020.29106.man

Cited by 16 publications

(5 citation statements)

References 13 publications

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“…But as with any powerful new technology, gene therapy’s capacity to positively influence humanity will depend on its accessibility. Some fear that gene therapy could exacerbate inequalities in the case that only the wealthy are able to access its gifts. , Though these arguments have often been applied to the more ethically challenging questions associated with germline gene editing, they are also relevant to somatic gene therapies. Equitable distribution of gene therapies will be important to create a future where everyone thrives.…”

Section: Discussionmentioning

confidence: 99%

Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies

2023

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Gene therapy has demonstrated enormous potential for changing how we combat disease. By directly engineering the genetic composition of cells, it provides a broad range of options for improving human health. Adeno-associated viruses (AAVs) represent a leading gene therapy vector and are expected to address a wide range of conditions in the coming decade. Three AAV therapies have already been approved by the FDA to treat Leber’s congenital amaurosis, spinal muscular atrophy, and hemophilia B. Yet these therapies cost around $850,000, $2,100,000, and $3,500,000, respectively. Such prices limit the broad applicability of AAV gene therapy and make it inaccessible to most patients. Much of this problem arises from the high manufacturing costs of AAVs. At the same time, the field of synthetic biology has grown rapidly and has displayed a special aptitude for addressing biomanufacturing problems. Here, we discuss emerging efforts to apply synthetic biology design to decrease the price of AAV production, and we propose that such efforts could play a major role in making gene therapy much more widely accessible.

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Section: Discussionmentioning

confidence: 99%

Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies

2023

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“…In September 2020, the U.K. Royal Society and the U.S. Academies of Sciences and Medicine released “Heritable Human Genome Editing.” 5 , 39 And in July 2021, the World Health Organization's Expert Advisory Committee on Developing Global Standards for Governance and Oversight of Human Genome Editing released two reports – regarding both the scientific aspects of this field. 40 , 41…”

Section: Recent International and Intranational Discussionmentioning

confidence: 99%

Governing Heritable Human Genome Editing: A Textual History and a Proposal for the Future

2021

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Heritable human genome editing (HHGE) has become a topic of intense public interest, especially since 2015. In the early 1980s, a related topic—human genetic engineering—was the subject of sustained public discussion. There was particular concern about germline genetic intervention. During the 1980s debate, an advisory committee to the Director of the National Institutes of Health (NIH)—the Recombinant DNA Advisory Committee (RAC)—agreed to provide initial public review of proposals for deliberate introduction of DNA into human beings. In 1984 and 1985, the RAC developed guidelines for research involving DNA transfer into patients. The committee also commented on the possibility of deliberately altering the human germline. We track the textual changes over time in the RAC's response to the possibility of germline genetic intervention in humans. In 2019, the NIH RAC was abolished. New techniques for genome editing, including CRISPR-based techniques, make both somatic and germline alterations much more feasible. These novel capabilities have again raised questions about oversight. We propose the creation of a new structure for the public oversight of proposals to perform HHGE. In parallel with a technical review by a regulatory agency, such proposals should also be publicly evaluated by a presidentially appointed Bioethics Advisory Commission.

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“…The Delphi method provided the empirical evidence which laid the basis for Human Genome Editing Report, published in September 2020, which suggested a translation route for limited approval of germline editing under special circumstances and subject to a set of criteria. The study involved about three dozen experts from the field of genome editing, medicine, bioethics, law and related fields [26].…”

Section: Methodological Tools Used In Interdisciplinary Social and Hu...mentioning

confidence: 99%

Human Genome as an Object of Interdisciplinary Socio-Humanitarian Research and a System of Appropriate Methodological Means

Fetyukov

2022

SHS Web Conf.

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The research is based on a comprehensive analysis of scientific articles devoted to the problems of obtaining, storing and using genomic information about a person. As a result of the analysis, the author put forward a hypothesis about the interdisciplinary interaction of social sciences and humanities in the process of research, the object of which is the human genome. The study tests the proposed hypothesis on the basis of the disclosure of its subject - the human genome as an object of interdisciplinary social and humanitarian research and the system of methodological means corresponding to them. During the research, the author relied on the general philosophical means of the methodology of science (categories of materialistic dialectics, foundations, conditions and principles of scientific knowledge), the systemstructural method and general logical methods (analogy, synthesis, analysis) used in socio-humanitarian studies. As a result of the study, it was concluded that the special significance of the consequences of the study and use of human genome for society and each individual personally determines the a priori social and humanitarian interdisciplinary connections, due to which human genome becomes the object of interdisciplinary social and humanitarian research. The analysis of these studies allowed revealing their subject orientation towards the social regulation of genomic research, the legal protection of genomic information and the focus on standardization (and in this sense, foreseeability) of research on human genome and the use of their results. At the same time, the use of methodological means of three levels is traced: philosophical and methodological analysis, general scientific methodological principles and a special scientific methodological approach.

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Reactions to the National Academies/Royal Society Report onHeritable Human Genome Editing

Cited by 16 publications

References 13 publications

Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies

Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies

Governing Heritable Human Genome Editing: A Textual History and a Proposal for the Future

Human Genome as an Object of Interdisciplinary Socio-Humanitarian Research and a System of Appropriate Methodological Means

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