New Book, “Run to the Light,” Chronicles a Journey with Batten Disease

  On Sunday, August 19, Taylor King will turn 20. On November 1, Laura King Edwards’s inspiring book “Run To The Light” will be published, by Bedazzled Ink. The memoir is an astonishing tale of

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

Gene Therapy: Hoopla and Hurdles

0000-0002-8715-2896 Today I’m giving an invited lecture at Georgia College, “Gene Therapy: A Forever Fix”? I’ve given the talk many times, since my book The Forever Fix was published in 2012, but this is the first

Will Short Term and Long Term Treatments for Single-Gene Diseases Survive?

0000-0002-8715-2896 Two weeks and several political disasters ago, the House of Representatives passed the American Health Care Act of 2017, and soon lists of “pre-existing conditions” festooned news feeds. We all ticked off a few.

Hannah’s 2016: From Curling Toes to Gene Therapy

CaptureEleven-year-old Hannah Sames can still curl her toes, just barely. But time is running out. If Hannah can move her toes for a few more weeks, until she becomes the fourth child in a clinical

Precision Medicine Initiative – Ricki’s Pick for Breakthrough of the Year

NHGRI-85326-588x320‘Tis the season for Science magazine to name their Breakthrough of the Year, a designation that typically irks me because it implies that science happens suddenly and we all know that of course it doesn’t.

Midsummer Updates at DNA Science

Summer is half over, so I thought I’d update a few posts. EMAN IN LIBERIA A year ago, I frantically wrote about my young friend in Liberia, Emmanuel Gokpolu, and his pleas to help stop Ebola. Emmanuel and his loved ones … Continue reading »

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“Saving Eliza” Campaign Helps Another Child

Valerie Byers had long suspected that her son Will’s diagnosis of autism was wrong. So when she saw a clip on the homepage of the Today Show about a little girl named Eliza, in late February, she knew instantly that 5-year-old … Continue reading »

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Hannah’s Hair – Why Traits Matter

It’s an unacknowledged law of nature that whatever the texture of a girl’s hair, she wants the opposite. For years I wrapped my tangles around soup cans and around my head, squished it under irons, and subjected it to stinky … Continue reading »

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Gene Therapy for Blindness Works!

The news this week presented at a major vision conference and published in The New England Journal of Medicine about gene therapy to treat childhood blindness paints an unnecessarily grim picture. Because I wrote a book about it and know affected families, … Continue reading »

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