Genetic Linkage
Gene Therapy Changes the Brain
February 8, 2012
It isn’t often that a brain scan chokes me up, but this one did. The fMRI shows area 17 of the visual cortex coming to life in a woman born with Leber congenital amaurosis type 2 (LCA2). She’s part of the very same gene therapy clinical trial chronicled in my upcoming book The Forever Fix: Gene Therapy and the Boy Who Saved It. The symbolic boy is Corey Haas, who, four days after gene therapy in 2008 at age 8, screamed when he saw the sun at the Philadelphia zoo, his shadow world suddenly brightened. Read More
2 Comments
Gene Therapy and the 10,000-Hour Rule
January 4, 2012
“Breakthroughs” in biomedicine are rarely that – they typically rest on a decade or more of experiments. Consider gene therapy.
I just unearthed an article from the December 1990 issue of Biology Digest, "Gene Therapy." I wrote it a mere two months after the very first gene therapy experiment, the much-publicized case of 4-year-old Ashi DeSilva, Read More
I just unearthed an article from the December 1990 issue of Biology Digest, "Gene Therapy." I wrote it a mere two months after the very first gene therapy experiment, the much-publicized case of 4-year-old Ashi DeSilva, Read More
Gene Therapy Subverted in New Dystopian Novel, “When She Woke”
December 30, 2011
I don’t usually take too kindly to the evil geneticist stereotype in fiction, but I can’t resist a good dystopian novel. "When She Woke," by Hillary Jordan, is the perfect book Read More
Respiratory Replacement Parts -- Thanks to Stem Cells
November 29, 2011
We humans might not be able to regrow a leg, as can a cockroach or salamander, or regenerate a missing half, like a flatworm, but our organs can replenish themselves – thanks to stem cells. Two new reports about opposite ends of the respiratory system may pave the way for replacement breathing parts.
A 36-year-old Read More
A 36-year-old Read More
Gene therapy on fetuses?
November 4, 2011
Gene therapy is experiencing a renaissance, with many of the recent successes in children. For some conditions, the younger the child, the better the genetic correction, because affected tissues degenerate with time. This is the case for adrenoleukodystrophy (ALD), the “Lorenzo’s Oil” disease that strips the insulation from brain neurons. One goal of Read More
23andMe's Exome Sequencing and the Tenth Edition of My Textbook
October 1, 2011
It’s been a strange week. The tenth edition of my human genetics textbook was published, just as 23andMe announced that they now offer whole exome sequencing, for $999. Read More
"The Sighted Leading the Sighted"
August 3, 2010
Normally a 9-year-old boy cavorting with a big, shaggy dog isn’t anything unusual, but when Corey Haas grabbed the leash of 1-year-old Mercury last Saturday, it was a stunning sight. For both Corey and Mercury, a briard sheepdog, were born with the exact same form of hereditary blindness, Leber congenital amaurosis (LCA). And Read More
Creating Life and Curing Blindness
May 21, 2010
I’ve been at the American Society of Gene and Cell Therapy annual meeting this week, garnering tales for my book, tentatively entitled “The Forever Fix.” It is largely the story of 9-year-old Corey Haas, who was on his way to certain blindness when gene therapy performed at the Children's Hospital of Philadelphia in September 2008 Read More