Gene Editing Technology and Ethical Issues

Background

Five years ago University of California, Berkeley, biochemist Jennifer Doudna, and colleagues unveiled the gene-editing tool CRISPR (Science 2012). Doudna's lab, in fact, was engaged in a patent fight with the Harvard- and MIT-affiliated Broad Institute.

The patent case turns on the question of which researchers at the two institutions conceived of the most important CRISPR applications first. UC says it's a team at its Berkeley lab headed by Jennifer Doudna, with the collaboration of Emmanuelle Charpentier, now of Germany's Helmholtz Centre for Infection Research. Broad's claim is based on the work of its researcher Feng Zhang.

The patent office changed its rules to a “first-to-file” basis from the old “first-to-invent,” in 2013, making the U.S. the last major country to do so. The idea was to end disputes that turned on minute interpretations of lab records or personal notes, with costs that arguably disadvantaged small inventors.

Last February 2017, the U.S. Patent Trial and Appeal Board ruled that although a team led by UC Berkeley structural biologist Jennifer Doudna had first laid claim to the use of CRISPR to cut DNA in a test tube, the use of the method on human cells by molecular biologist Feng Zhang’s team at the Broad was still an advance.

However, a decision from the European Patent Office (EPO) in January 2018 revoked the patent from the Broad Institute of MIT and Harvard University because the Broad did not meet EPO requirements to establish that its researchers were the first to use CRISPR in eukaryotes. The patent granted to the Broad for fundamental aspects of the technology is one of several of its patents facing opposition in Europe.

Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies can add, alter, or genetic material at particular locations in the genome. Several approaches to genome editing have been developed. Once the DNA is cut, researchers use the cell's own DNA repair machinery to add or delete pieces of genetic material, or altering the DNA by replacing an existing segment with a customized DNA sequence.

Genome editing is of great interest in the prevention and treatment of human diseases. It is being explored in research on a wide variety of diseases. It also holds promise for the treatment and prevention of a more complex disease, such as human immunodeficiency virus (HIV) infection.

Figure 1.