Eleven years ago, gene therapy — where defective genes are snipped out of DNA and replaced with healthy ones — became a household name. A landmark paper proved that scientists could precisely manipulate DNA in ways previously thought unimaginable using CRISPR-Cas9, an editing tool adapted from the immune system found in some bacteria. Almost overnight, the idea of designer babies, kill-switch mosquitoes, and cancer-off buttons stormed into mainstream imagination.
Since then, gene therapy has experienced a complete renaissance, culminating this past November and early December when , set in a world where genetic engineering and socioeconomic status go hand in hand. Both before and after the infamous incident involving Chinese scientist He Jiankui creating the world’s first CRISPR-edited human babies with a gene for HIV immunity, there have been strict worldwide regulations on any gene editing research involving embryos.
But scientists like Mitalipov are looking into using CRISPR to potentially adjust an embryo’s risk for disease. His own research at OHSU involves gene-editing germline cells — or reproductive cells that pass on genetic information to the offspring — in what Mitalipov calls “IVF gene therapy.”
He says such a technique could help improve the success of embryo implantation during IVF by creating stronger, more viable embryos. Mitalipov acknowledges, however, that there needs to be a robust regulatory framework in place before we can ever truly consider genetically engineered babies.
“In terms of regulation, we have to focus only on those 10,000 gene defects we know today that cause human disease,” says Mitalipov. “It could be easily mandated that gene therapies in embryos have to be towards severe disease in children.”
But that, and the rest of gene therapy’s optimistically bright future, remains hedged with abundant yet much-warranted caution.