Gene Therapy

Biomarker Tests for Molecularly Targeted Therapies


The Key to Unlocking Precision Medicine NEJM   An IOM committee focused on biomarkers for molecularly targeted therapies has issued recommendations about regulatory, reimbursement, and clinical practice issues, in order to guide precision medicine in advancing the care of patients with cancer and other diseases. This article was published on June 1, 2016, at Disclosure forms […]

The Lab Results Are In: Genes Might Be to Blame for Retrievers’ Obesity


  If you’ve ever had a Labrador retriever, you know about one of the breed’s notable traits: an unrelenting appetite. The dogs will devour anything from socks to rocks, and given the chance, they can be prone to eat themselves into obesity. By one estimate, nearly 60 percent of all Labradors are overweight or obese. […]

Gene drives aren’t ready for the wild, report concludes


Mosquitoes and other organisms normally have a 50 percent chance of passing along a gene to an offspring. A gene drive copies and pastes itself into chromosomes from both parents, ensuring it gets passed on more often. Mosquitoes and invasive species can rest easy — for now. Genetic technology called gene drives could wipe out […]

First Gene Therapy for Children is Approved in Europe


Radical treatment for rare ‘bubble boy’ disorder has a 100% survival rate The European Commission has given approval for British drug firm GlaxoSmithKline to produce the world’s first gene therapy treatment for children. GSK will produce Strimvelis, the first stem cell gene therapy to treat patients with severe combined immunodeficiency caused by adenosine deaminase deficiency […]

Genetically engineered immune cells are saving the lives of cancer patients


Immune Engineering – That may just be the start The doctors looking at Layla Richards saw a little girl with leukemia bubbling in her veins. She’d had bags and bags of chemotherapy and a bone marrow transplant. But the cancer still thrived. By last June, the 12-month-old was desperately ill. Her parents begged—wasn’t there anything? […]



On São Miguel Island, in the Azores archipelago in the middle of the North Atlantic, lives a curious Brazilian expat. Known as Pontoscolex corethrurus, this tropical worm cannot tolerate the isle’s temperate climate. And yet it survives there in two areas: in artificially warmed pineapple greenhouses and the soil within an active volcano. Read more here

Scientists Talk Privately About Creating a Synthetic Human Genome


Sixty trays can contain the entire human genome as 23,040 different fragments of cloned DNA. Scientists are now contemplating the fabrication of a human genome, meaning they would use chemicals to manufacture all the DNA contained in human chromosomes. The prospect is spurring both intrigue and concern in the life sciences community because it might […]

Genome study offers new hope for children with rare diseases


An international team involving University of Queensland researchers has used advanced genome sequencing to diagnose 30 patients with unresolved rare diseases. The patients were among 70 people with disorders of the brain’s white matterand were examined using whole exome sequencing (WES)—a method that looks at all the genes in a person’s genetic code at once. […]

Breakthrough DNA Editor Born of Bacteria


On a November evening last year, Jennifer Doudna put on a stylish black evening gown and headed to Hangar One, a building at NASA’s Ames Research Center that was constructed in 1932 to house dirigibles. Read more here.

Should we bring extinct species back from the dead?


Earth is in the midst of its sixth mass extinction: Somewhere between 30 and 159 species disappear every day, thanks largely to humans, and more than 300 types of mammals, birds, reptiles, and amphibians have vanished since 1500. Read more here.

CRISPR Patent Outcome Won’t Slow Innovation


Last week a panel of judges at the U.S. Patent and Trademark Office in Alexandria, Virginia, heard arguments as to who should own the rights to the century’s biggest biotechnology invention to date, a precise gene-editing system called CRISPR-Cas9 that has the potential to treat serious human genetic disorders and create designer crops that resist […]

Oral Arguments in CRISPR Patent Fight Slated for Today

Oral Arguments in CRISPR Patent Fight Slated for Today

On Tuesday morning, the CRISPR patent dispute reaches a much-awaited milestone: the case’s first and only oral arguments, slated to last less than an hour for a patent potentially worth billions of dollars. The hearing is open to the public, and it’s sure to attract the attendance of dozens of lawyers, company executives (Novartis has confirmed […]

Pop Star D.A. Wallach Champions Genetic Testing At D.C. Conference, But Others Remain Wary

Pop Star D.A. Wallach Champions Genetic Testing At D.C. Conference, But Others Remain Wary

At the recent HL7 Genomics Conference in Washington, D.C., the audience sat in rapt attention as pop musician-cum-venture capitalist D.A. Wallach shared, via Skype, the highly personal story of his wife Liz’s experience with contemporary clinical genetics. After her mother’s cancer diagnosis, Liz sought testing and discovered she carried a specific genetic mutation resulting in Lynch […]

How the DNA Revolution Is Changing Us

How the DNA Revolution Is Changing Us

If you took a glance around Anthony James’s office, it wouldn’t be hard to guess what he does for a living. The walls are covered with drawings of mosquitoes. Mosquito books line the shelves. Hanging next to his desk is a banner with renderings of one particular species—Aedes aegypti—in every stage of development, from egg […]

Gels to Repair Hearing Loss? Drugmakers Try New Ear Treatments

Gels to Repair Hearing Loss Drugmakers Try New Ear Treatments

Sarah Povey spent her youth at rock concerts, sometimes going out five or six nights in a row, soaking up the live sound from nearby speakers. By her late 20s, she was diagnosed with severe tinnitus, a ringing in the ear that makes it hard to listen to any music at all. “I was very, […]