Genetic Engineering & Biotechnology News

DEC 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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Page 64 of 69 | DECEMBER 2017 | 31 and donor designs are checked first at the pooled level to determine which ones are the most efficient; population events as low as 0.2% can be determined. GEiC also uses deep sequencing for genotyping animals modified with CRISPR/Cas9. Targeted deep sequencing at the loci of interest indicates what fractions of alleles in a mosaic animal have the desired modification. NGS will continue to play a role as genome editing evolves. Not only can the technology contribute to better experimental design, it can also play a role in identifying off-target effects. In a therapeutic situation, millions of cells are taken out, edited, and replaced. Whatever combination of gRNA and Cas9 combination is used, the chance of having an off-target event in a mil- lion cells is high. Some combinations, however, may pose a higher risk of off-target events than others. Whole-genome sequencing can pro- vide enough coverage of the genome to help determine if a therapy is safe. Mul- tiple reads of each allele can indicate where off-target effects are occurring. Making Policies Work has commenced on the genetic modification of human em- bryos—both nonviable (recently) and viable (more recently). It has highlighted the complexity of social, ethical, and policy decisions. "This a difficult area to define and fully comprehend," maintains Dr. Friedmann. "It involves not only legitimate and ethically acceptable forms of human genetic modification for medical purposes and disease control, but also points to ethically hazardous applications and potential policy disasters." Of particular con- cern are potential human enhance- ment and eugenic applications. "Nontherapeutic human genetic modification is developing with such speed that it is worrisome that human society may not be prepared for the moral, ethical, and policy dilemmas that it poses," Dr. Friedmann contin- ues. "What is a legitimate target for genome editing? Who will identify those targets and to what end?" As human genetic enhancement becomes increasingly feasible, we sometimes hear arguments that we are morally obligated to use any and all of the tools at our disposal to better humanity. Such arguments usually fail to clarify what is meant by better, who is going to be empowered to make such judgements, and how genetic and evolutionary harm may be avoided. If a gene is manipulated without totally understanding its interactions with other genes, then there is a chance of doing serious genetic harm. Legislators and policy makers are going to be faced with technical and policy issues for which they may not be adequately prepared. Perhaps it is time to resurrect a version of the Office of Technology Assessment (OTA), which served the U.S. Con- gress from 1972 to 1995. The OTA provided congressional members and committees with objective, nonpar- tisan, and authoritative analysis of the complex scientific and technical issues of the late 20th century. With- out access to updated information in such an explosively developing field, policy makers cannot be expected to make wise and enlightened deci- sions. B E S T O F C R I S P R 2 017

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