By Associated Press

After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.

by Emily Mullins

The first clinical trials are slated to begin in the U.S. and Europe while others are stalled.

by Laurie McGinley

Jay Konduros used to rush home several times a year after accidentally cutting or bumping himself. There he would inject himself with refrigerated blood-clotting factor to prevent internal bleeding and extensive bruising and swelling. “I was walking on eggshells all the time,” said the former aerospace engineer who has a blood disorder called hemophilia B.

by Christopher Vaughan

Stanford University School of Medicine researcher Matthew Porteus, MD, PhD, was one of three expert witnesses who spoke at a U.S. Senate committee hearing on the CRISPR gene editing technology this week.

by Fan Liu

Associate Professor of Pediatrics Matthew Porteus was recently awarded a $5.2 million grant from the California Institute of Regenerative Medicine (CIRM) to develop cutting-edge sickle cell anemia therapies.

by Jessica Zhang

Stanford Medicine will open a new Center for Definitive and Curative Medicine (CDCM) to treat people with genetic diseases using stem cells and gene therapies. The center is a joint-initiative with the school of medicine, Stanford Health Care and Stanford Children’s Health.

By Kate Marusina, PhD

Using CRISPR to Produce Better Disease Modelling

By Becky Bach

Good news for people suffering from genetic diseases and for those who could be helped with stem cell therapies. This week, Stanford announced the creation of the Center for Definitive and Curative Medicine, a new center that aims to bring life-changing advances to millions of patients.

By Erin Digitale

New gene-editing techniques are making it easier, cheaper and more efficient to rewrite the DNA of living organisms. But these methods — such as CRISPR — raise challenging questions about the ethics of editing the human genome.

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 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

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.