New Treatment for Phenylketonuria (PKU) Clears Brain Fog

In the 1959 novella Flowers for Algernon by Daniel Keyes (and the 1968 film Charly), 32-year-old Charlie Gordon, a janitor at a New York City bakery, undergoes experimental surgery that has boosted the intelligence of a

Gene Therapy for Myotubular Myopathy: Early Signs of Success!

Parents cherish developmental milestones, from a newborn’s grip of an offered finger; to an infant’s holding her head up the first time; to rolling over, creeping, and crawling; then to standing, cruising, and finally walking.

French Anderson, “Father” of Gene Therapy, Released From Prison

  Tonight French Anderson was released from state prison in California, after serving nearly a dozen years for “inappropriate touching and medical exams” of a girl, from 1997 to 2001, starting when she was ten.

Celebrating The Moms of Gene Therapy

As Mother’s Day and the annual meeting of the American Society of Gene and Cell Therapy approach, I’d like to update the stories of some of the moms I’ve written about, and introduce a new

What’s a “Variant of Uncertain Significance?” A VUS?

  Seven words someone taking a genetic test doesn’t want to hear: “You have a variant of uncertain significance.” A VUS. Instead of a yes or no answer – a gene has a mutation or

Experimental Drug Extends Survival in Progeria

A report from a clinical trial for a drug to treat the rapid-aging disorder progeria, published in this week’s Journal of the American Medical Association, offers hope for families with the ultra-rare genetic condition. Old Before

Tilapia: Freak Farmed Fish or Evolutionary Rock Star?

  By Ricki Lewis Posts are appearing on my Facebook feed warning against the dangers of eating tilapia. So I decided to do  a little research. My dad was a seafood wholesaler at the Fulton

Don’t Tell Me My DCIS Isn’t Cancer! | #AACR18

By Ricki Lewis “DCIS isn’t really cancer. You have nothing to worry about,” said my oncologist confidently. “Then why am I having a mastectomy in four days?” I blurted. “DCIS doesn’t spread. So it isn’t

Jackass Genomics – Did Donkeys Arise from an Inverted Chromosome?

0000-0002-8715-2896 In the world of genome sequencing, donkeys haven’t received nearly as much attention as horses. But now a report on a new-and-improved genome sequence of Willy, a donkey (Equus asinus) jack born at the

A Hiccup in Gene Therapy Progress?

0000-0002-8715-2896 Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new drug