Abstract
Genome editing and the technologies that enable it have sparked public discussions as investments in research and development continue to soar. Available gene editing modalities are enabling far-reaching applications beyond heritable genome modifications, ranging from novel therapeutics and cancer immunotherapies to engineered crops and livestock. However, many challenges, both practical and ethical, still exist before genome editing technologies can be implemented. Existing legal frameworks, both national and international, are racing to keep pace with the development of gene editing technologies. Updating regulations on gene editing can help provide a framework under which scientists and the public operate. Shaping and implementing proper regulations will require engagement from all impacted stakeholders. Here, we present a comprehensive review of the current scientific and regulatory landscape of this field of gene editing in order to stimulate necessary conversations regarding future regulations in the broader community
Highlights
Genome editing and the technologies that enable it have sparked public discussions as investments in research and development continue to soar
I n recent years, genome editing technology has come to the forefront of therapeutic biotechnology development and has already begun to revolutionize medicine
CRISPR-based genome editing was first demonstrated in 2013 [1]–[4], there have been over a dozen clinical trials for CRISPR-enabled therapeutics, including treatments for blood disorders, cancers, and even childhood blindness [5, 6]
Summary
Characterization of in vitro Cas m2e0ch1a5n-pisrmesent Development of new CRISPR. vectors can be used both for ex vivo as well as in vivo delivery. AGN-151587, the CRISPR-based drug currently in in vivo clinical trials for Leber’s Congenital Amaurosis, is another notable example [81] Considering such successes, AAV remains a promising mode to deliver genome editing therapies and, is routinely used in research settings to do so in animal models of disease. In July of 2019, the advisory committee issued a statement condemning clinical application of germline genome editing until its implications have been thoroughly considered [122]–[124], effectively recommending a moratorium similar to those previously suggested [19, 23] This committee has further recommended development of a global framework using the guiding principles of transparency, inclusivity, fairness, social justice, and responsible scientific stewardship that can be scalable from local to international governments, can work in contexts with both tight and loose regulation of scientific practice, and is developed in collaboration with the widest possible range of stakeholders [122, 124]. Additional international initiatives have included creation of a global registry to track human genome editing research in both somatic and germline contexts [125, 126] in a hope to provide more public transparency to the process of human germline editing research
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