Category Archives: gene therapy
New Book, “Run to the Light,” Chronicles a Journey with Batten Disease
Celebrating The Moms of Gene Therapy
Posted by featured, gene therapy
inGene Therapy: Hoopla and Hurdles
Posted by featured, gene therapy
inWill Short Term and Long Term Treatments for Single-Gene Diseases Survive?
Hannah’s 2016: From Curling Toes to Gene Therapy
Posted by clinical trials, featured, GAN, gene therapy, giant axonal neuropathy, Hannah Sames, Lori Sames, NORD, physical therapy, Steven Gray, Xenokeryx
inPrecision Medicine Initiative – Ricki’s Pick for Breakthrough of the Year
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
The post Midsummer Updates at DNA Science appeared first on PLOS Blogs Network.
“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
The post “Saving Eliza” Campaign Helps Another Child appeared first on PLOS Blogs Network.
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
The post Hannah’s Hair – Why Traits Matter appeared first on PLOS Blogs Network.
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
The post Gene Therapy for Blindness Works! appeared first on PLOS Blogs Network.