By Greta Lorge

Stem cell research holds the promise of a sea change in medicine. At Stanford, momentum is building.

BREAKTHROUGHS, BRIEFLY

School of Medicine Research led by associate professor Matthew Porteus, MD ’94, PhD ’94, may advance to human gene-therapy trials to treat sickle cell disease by next year. That, anyway, is the timetable Porteus is hoping for following his team’s promising use of the gene-editing technique known as CRISPR to correct the gene mutation that produces misshapen—sickle-shaped—red blood cells.

By Emily Mullin

Scientists have solved fundamental problems that were holding back cures for rare hereditary disorders. Next we’ll see if the same approach can take on cancer, heart disease, and other common illnesses.

Guest host Anthony Brooks

Growing support for human gene-editing. We’ll look at new breakthroughs and the ethical debate

Those in the know call CRISPR “one of the greatest life science inventions ever.” It has sparked a headline-grabbing patent fight worth billions of dollars. But more importantly, it has revolutionized the ability to make precise changes to human DNA, opening the door to revolutionary ways to treat disease – but also to ethical questions about engineered designer babies. This hour On Point, the brave new world of human DNA editing and CRISPR.

By Erin Digitale

Using stem cells and gene therapy to treat or cure disease may still sound like science fiction, but a scientific meeting here last week emphasized all the fronts on which it is moving closer and closer to fact.

By Allison Proffitt

The Chan Zuckerberg Biohub has named its first cohort of 47 investigators from the University of California, Berkeley, Stanford University and the University of California, San Francisco.

The grants to Stanford researchers target stem cell-based therapies for autoimmune disorders, liver disease and cystic fibrosis.

Three researchers from the School of Medicine have been awarded grants by the governing board of the California Institute for Regenerative Medicine to promote the discovery of potential stem cell-based therapies.

By Erin Digitale

“One clinical picture can have so many different underlying causes,” said pediatric hematologist Katja Weinacht, MD, PhD. When a patient’s immune system is attacking the body’s own red blood cells, for instance, non-specific treatments like steroids may not be enough to put immune disease into remission, Weinacht explained. And the treatments’ side effects can exact a heavy price, especially from young patients.

By Luke Dormehl

When you read about initiatives like Stanford University’s new Laboratory for Cell and Gene Medicine, it’s difficult to think of too many other places on Earth where more exciting — and potentially life-changing — work is being done.

By Lisa M. Krieger | lkrieger@bayareanewsgroup.com | Bay Area News Group

Cells made in new Laboratory for Cell and Gene Medicine combat cancer and genetic disease

PALO ALTO — To the list of cool new things made in Silicon Valley, add this: Life, improved.

By Kerry Grens

Researchers say they have sufficient in vitro and animal data to apply for human testing.

In another demonstration of the clinical utility of CRISPR, researchers have corrected the gene underlying sickle cell disease in human cells. In a Nature paper published Monday (October 7), the team reported that the treated hematopoietic stem cells survived for at least 16 weeks when transplanted into the bone marrow of mice.

By Lisa M. Krieger | lkrieger@bayareanewsgroup.com | Bay Area News Group

Success is part of an accelerating research movement made possible using the new technique, called CRISPR-Cas9

By Jennie Dusheck

In the 1980s, molecular biologists conceived the idea of gene therapy, in which a genetic disease might be cured by replacing the mutated disease-causing gene variant with a functioning version of the same gene.

By Lisa M. Krieger | lkrieger@bayareanewsgroup.com | Bay Area News Group

STANFORD — Some day — when your heart needs healing, your bones need bonding or your skin needs stitching — clusters of new cells now growing in a Stanford University lab could offer a fix.

By Antonio Regalado

Rewriting Life Gene Therapy’s First Out-and-Out Cure Is Here A gene therapy for an inherited immune disease completes a 27-year journey.

A treatment now pending approval in Europe will be the first commercial gene therapy to provide an outright cure for a deadly disease.

By Lisa M. Krieger | lkrieger@bayareanewsgroup.com | Bay Area News Group

STANFORD — Tiny vials of recently repaired blood cells are thriving in a Stanford incubator, proof that a powerful new gene-editing technique is fixing errant genes that cause so much human suffering.

By Mike Antonucci

Sudden huge advances in genetic engineering have scientists mobilizing to avert an ethical maelstrom.

By Krista Conger

The CRISPR news just keeps coming. As we’ve described here before, CRISPR is a breakthrough way of editing the genome of many organisms, including humans — a kind of biological cut-and-paste function that is already transforming scientific and clinical research. However, there are still some significant scientific hurdles that exist when attempting to use the technique in cells directly isolated from human patients (these are called primary cells) rather than human cell lines grown for long periods of time in the laboratory setting.

By Christopher Vaughan

It has been a momentous month for Stanford researcher Maria Grazia Roncarolo, MD. Following decades of research in Roncarolo’s lab and the clinic, pharmaceutical company Glaxo SmithKline has applied for final approval by European Medicines Agency (EMA) of a treatment she developed to cure a deadly childhood immune disorder. If approved by the EMA, which is Europe’s equivalent of the U.S. Food and Drug Administration (FDA), the treatment would be the first gene stem cell therapy to be granted approval by a major medical regulatory agency.

By Christopher Vaughan

Academic institutions are in a much better position than pharmaceutical companies to make the best decisions about which therapies deserve further development. That was the underlying message from a pair of Stanford researchers at a panel on stem cell science at last weekend’s Association of Health Care Journalism 2015 conference.

By Christopher Vaughan

At the Childx conference last week there was a great deal of optimism that stem cell and genetic therapies are about to have a huge impact on many childhood diseases. “It’s not just science fiction anymore,” Matthew Porteus, MD, PhD, told the audience. “We can correct mutations that cause childhood disease.”

By Erin Digitale

Next month’s inaugural Childx conference will bring a diverse group of experts to Stanford to discuss big challenges in infant, child and maternal health. Today, in a new 1:2:1 podcast interview, stem cell and gene therapy expert Maria Grazia Roncarolo, MD, provides an interesting preview of a once-controversial area of research that will be featured at the conference.