Delaney Van Riper suffers from a mutation in a single gene that leads to stumbling and weakness in her hands and feet. Â Through the use of the gene-editing technology CRISPR, Van Riper, a student at UC Santa Cruz, hopes to overcome her condition.
ONVENTIONAL SURGERY canât help patients such as Delaney Van Riper, a 19-year-old college student with an independent spirit, love of literature and progressive neurological disease. But gene surgery might. In recent weeks, a molecular scalpel began to make test cuts on a troubling mutation in Van Riperâs cells that causes stumbling and weakness in her hands, in hopes of allowing a healthy gene to take over and multiply. While the research is preliminary â her DNA is being changed in the lab, not her body â itâs a step toward fulfilling the therapeutic promise of gene editing, offering a one-time procedure to cure devastating genetic disorders and potentially helping millions of people around the planet. The pioneering research is underway at Dr. Bruce Conklinâs lab at the nascent Genome Surgery Initiative, part of a broader Bay Area-based effort to see if our genetic blueprint can be fixed as efficiently and effectively as bones, hearts and other parts of the human body. âItâs anatomy that weâre cutting out,â explained Conklin, the UCSF/Gladstone Institutes researcher who co-conceived the Initiative and dreams of making gene surgery widely available to the public. âItâs just very small anatomy.â
Dai Sugano/Bay Area News Group Dr. Bruce Conklin, a senior investigator at Gladstone Institutes, sits in front of an image of the molecular structure of a disease-causing protein that his team is targeting for removal through genetic surgery.
The precise cutting tool, called CRISPR-Cas9, alters the genetic sequences in cells. It isnât the first gene-editing method. But it is much faster, cheaper, easier and more accurate than earlier versions. âItâs like the Model T â not the first car but the one that changed the world,â according to Hank Greely, director of the Center for Law and the Biosciences at Stanford Law School. Its discovery in 2013 by UC Berkeleyâs Jennifer Doudna galvanized the medical community â and now, only five years later, it is moving out of test tubes and toward testing in humans, with clinical trials for various diseases slated to start next year. But itâs no one-trick pony. Different strategies can be enlisted for different disorders. In some surgeries, such as Delaneyâs, CRISPR merely cuts out a bad gene. For diseases such as sickle cell, it must cut, correct and replace. Thereâs no guarantee that CRISPR will cure Delaney of the disease that causes her to stumble when she walks or struggle when she opens a bag of shredded cheese. Things that work perfectly in a test tube often fail in the human body. There are concerns about elevating the risk of cancer or cutting DNA in the wrong place. And CRISPR canât fix medical problems caused by multiple genes, such as heart disease or diabetes.
Randy Vazquez/ Bay Area News Group Van Riper opens a bag of cheese with a pair of scissors. âI can feel myself telling my body to, like, grip it a certain way or use certain muscles but it doesnât actually happen,â she said.
But hereâs the dream: If research succeeds, then one day â not too far away â doctors could build a common âpipelineâ of gene therapies, creating the efficiencies and economies of scale needed to cure the estimated 6,000 to 8,000 single-gene disorders afflicting 350 million people around the world. In support of that vision, Conklin and other innovative Bay Area thinkers are envisioning a path-breaking role for a future Genome Surgery Initiative â a collaboration among UCSF, UC Berkeley and, perhaps, Stanford â that would establish the Bay Area as a center of genetic excellence, spinning off lucrative new innovations. âWeâve always thought about genetic disease as something which is incurable, something that youâre born with,â said Conklin. âWeâve never really thought about it as something that we could actually cut out or repair.â âWith this new editing tool,â he said, âwe can think about how to do this for the very first time.â â
Nicnivian on June 25th, 2018 at 04:59 UTC »
I have pretty advanced RP and am hopeful this technology can help others in the future. (I don't hold much hope for myself and I'm okay with that)
s0v3r1gn on June 25th, 2018 at 02:35 UTC »
One version dubbed a “cancer vaccine” just received approval for human trials.
Phedis on June 25th, 2018 at 00:08 UTC »
I was all excited until I read "it can't be used to cure diabetes". 😥