Personal Genetics Education Project

In the News: Developing personalized therapy using animal models

In a recent article in The Atlantic, journalist Ed Yong writes about efforts by researchers in Wisconsin to enlist the help of pigs in hopes of advancing personalized medicine. The scientists are planning to use genome editing to make “personalized piglets” for finding potential treatments for children with a hereditary condition called neurofibromatosis type 1 (NF-1). Children with this condition have mutations in their neurofibromin 1 gene; more than a thousand different genetic variants have been identified in this gene. Symptoms of NF-1 range from “café au lait” spots on their skin (patches with darker color than the surrounding skin), learning disabilities, to tumors in skin and nerves that can cause disfigurement and even death.

The Wisconsin researchers decided to use pigs as animal models for studying NF-1 because, in terms of size and anatomy, pigs are more similar to humans than more commonly used models like mice or rats. The hope is that, by studying NF-1 in pigs, it would be possible to discover treatments that have a higher chance of success in humans. By using the genome editing tool CRISPR to make patient-specific mutations in the pigs’ genomes, scientists also hope that they will be able to devise personalized treatment that works for individual patients.

This news story exemplifies one of the ultimate goals of personalized medicine – studying a disease the way it develops in a person and tailoring therapies to that individual’s genetic makeup. But this approach is in early stage, with a lot more research still required.

In 2013, Esquire magazine did an in-depth story on Stephanie Lee, a woman from Mississippi who developed colon cancer, whose case was taken up by a research team in New York City. By recreating, in fruit flies, the genetic mutations associated with Stephanie’s cancer, the scientists hoped to find a personalized drug treatment that will work for her. At the end, even though some promising leads were found using this approach, Stephanie did not survive long enough, in a healthy-enough state, to receive a personalized treatment; she passed away in 2015. Stephanie’s story highlights the “growing pains” of a field still in its infancy. But it also reminds us that, for all the technical advances and molecular wizardry promised by personalized medicine, perhaps it is – in the words of Tom Junod, one of the writers of the Esquire story – the “human and ancient” miracles of loyal friends and caring physicians that still matter the most for patients.

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