I don’t think I’ll ever tire of covering new ways to treat genetic diseases that I’d always thought hopeless. Teamed with expanded…
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post Two New Ways to Treat A Deadly Disease: Spinal Muscular AtrophyRead more
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post What Should Gene Therapy Cost?Read more
By the time that the FDA’s advisory committee gave a near-instantaneous and unanimous thumbs-up for gene therapy to treat a form of hereditary…
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post As Blindness Gene Therapy Nears FDA Finish Line, A Shout-Out to Activist FamiliesRead more
On a spectacular September Sunday in 2008, 8-year-old Corey Haas, a cane in one hand and his mother’s hand in the other…
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post Brain Cancer in Kids: Tailoring Treatment Based on MutationsRead more
I’ll admit it, I was sucked in. “Sharon was given a few months to live if her cancer wasn’t treated,” somberly intones…
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post Poliovirus To Treat Brain Cancer: A Curious ChronologyRead more
Certain things have a natural order. Breakfast before lunch. Infancy before adolescence. Autumn before winter. So I was surprised to read an…
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post White Supremacy: The Dark Side of EugenicsRead more
Whenever I work on a new edition of my human genetics textbook and reach the section on eugenics, at the end of…
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post Gene Therapy and September ScenesRead more
Today I’m giving an invited lecture at Georgia College, “Gene Therapy: A Forever Fix”? I’ve given the talk many times, since my…
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post A Genetic Disease With a Domino Effect: Multiple Sulfatase DeficiencyRead more
Willow is a beautiful name. Meaning slender and graceful, like the tree, it evokes images of a little girl running through the…
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post Were Ancient Humans Healthier Than Us?Read more
A curious thing happened when researchers at Georgia Tech used modern human genome sequences to look back at the possible health of…
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post Genome Cloaking Preserves Privacy While Enabling DiagnosisRead more
Sequencing genomes to diagnose puzzling symptoms presents a conundrum: how to interpret whether a person’s genotype causes the syndrome without comparison to…
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post Are Stem Cell Companies Abusing ClinicalTrials.gov?Read more
I’m often asked about the safety of treatments that purport to inject stem cells into painful body parts. The reputation of stem…
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post Charlie Gard Post-Mortem: Could He Have Been Saved?Read more
Charlie Gard would have turned one year old tomorrow. Two days before the British infant died of a mitochondrial disease on July…