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5 May 2016AmericasTony Trippe

The world of gene editing patents

For more than 60 years researchers have explored gene therapy, now referred to as “gene editing”. Initial attempts in the 1950s utilised live vaccines to manipulate genetic sequences. More sophisticated methods began to emerge during the 1980s and early 1990s with endeavours to modify human DNA, including the first direct insertion of human DNA performed by French Anderson in September 1990.

The general goal of gene editing is to replace, or disrupt, defective genes in order to provide a therapeutic benefit to the patient. Many genetic-based diseases involve a multitude of genes with complicated interactions and expression scenarios that make direct intervention difficult to manage.

However, there are many diseases that appear to be affected by a single gene, including cystic fibrosis, haemophilia, muscular dystrophy, thalassaemia and sickle cell anaemia, and correction of these defective genes has been the primary focus of therapeutic intervention using gene editing.

Human disease interventions are only one use of this technology. The method is increasingly being used to add desirable characteristics to plants, and useful traits in bacterial species. It can be argued that due to the simpler genomic make-up of some of these organisms gene editing there has been more productive than in humans. While this may be the case it hasn’t stopped scientists from working with human embryos, including a well publicised attempt by Chinese scientists to use gene editing methods on non-viable embryos to correct a mutation that causes beta thalassaemia.

“While The Broad Institute and the University of California receive most of the press coverage in this space, the organisation with the highest number of inventions is Cellectis.”

While this initial attempt was unsuccessful it led to the planning of the International Summit on Human Gene Editing which took place in Washington, DC in December 2015. The collected scientists agreed to continue with basic research, but also stressed the need for appropriate legal and ethical guidelines.

Analysis

A handful of methods have come to define the most commonly used approaches to gene editing. All involve the use of nucleases: enzymes that can cleave DNA sequences for modifications. The various methods focus on ways these nucleases can be delivered to specific locations in the genetic sequences of the target organisms.

The most widely studied systems include:

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