CRISPR comes to the clinic

CRISPR technology is a simple yet powerful tool for editing genes. It allows researchers to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, such as those that cause CGD. 

CRISPR is an enzyme that cuts DNA very precisely and allows researchers to take out the broken gene a person was born with and replace it with a healthy one at the same place where the gene normally sits in a person’s genetic make-up (genome). This is in contrast with current conventional gene therapy technologies that insert a replacement gene into the genome at random sites. Gene editing, however, repairs the gene in its usual position so it continues to be regulated by the natural control elements that are already present.

In a fascinating and highly recommended article by Kendall Morgan, published in Genome Magazine, the potential of CRISPR to treat CGD and other conditions is described in more detail. The article features the stories of two families affected by CGD. The first story is of Lake Wright from the UK, who has received conventional gene therapy, and Rachel, his mum. The second is of US-resident Robert Karp, who has taken part in the early research studies on CRISPR at the National Institute of Health in the USA.

Posted February 2018