An application to conduct gene-editing research in human embryos, by the Francis Crick Institute in London, has been approved by the UK’s regulatory body, HFEA. The research using CRISPR/Cas9 tools, proposed by Dr. Kathy Niakan, may begin within months, subject to ethics approval. The proposed research to use CRISPR/Cas9 technology, entitled “Derivation of stem cells from human embryos: the development of human embryonic stem cell (hES) cultures, characterisation of factors necessary for maintaining pluripotency and specific differentiation towards transplantable tissues”, was discussed in detail by the HFEA committee and was approved for a period of 3 years. The decision contrasts with the more conservative approach adopted by the US Office of Science & Technology Policy in which an announcement in May 2015 stated that “altering the human germline for clinical purposes is a line that should not be crossed at this time”. Controversy over the potential use of CRISPR based gene editing has increased dramatically since the publication of a research paper by Chinese scientists reporting use of the technology on human embryos in April 2015.
CRISPR/Cas9 gene editing is the most recent technological advance in the field of gene editing and may have significant impact across a range of ocular disorders including the direct delivery to gene editing tools to ocular tissues to target specific gene pathways, and the preparation and production of stem cell treatments for a number of degenerative retinopathies. The immediate aims of Dr. Niakan’s research are to further understand human embryo development to improve IVF technologies, and to establish new human stem cells for therapeutic applications. In terms of therapeutic applications, Dr. Niakan’s research efforts will be directed at establishing “defined, animal product-free conditions for the derivation of pluripotent human embryonic stem cell lines, ultimately leading to Good Manufacturing Practice (GMP) compatible approaches” and, “to establish and characterize human extraembryonic stem cell lines.
In granting the license the Committee commented that “the use of human embryos is necessary because the proposed research aims to study the role of gene products in human embryos that are not present in mouse embryos at the same stage; moreover, human embryonic development is significantly different to that of animal model species in a number of respects and, additionally, not all functions required for preimplantation development can be modelled in embryonic stem cells. The Committee was also satisfied that the number of embryos which might be used in the research was justified by the PR (person responsible], and it was reassured by the detailed information from the PR about how embryos would be used in a limited way at each stage of the intended research”.