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Glenn Nichols, surrounded by his hospice team. The author is in yellow.

Genetic Linkage

Excerpt about Canavan disease from "The Forever Fix: Gene Therapy and the Boy Who Saved It"

December 19, 2012

Tags: gene therapy, Canavan disease

Max Randell smiles at his middle school graduation.
The excerpt below supplements the blog entry that is beneath it and also at Public Library of Science. It is from The Forever Fix: Gene Therapy and the Boy Who Saved It, (St. Martin's Press, 2012).


I sat at a table with Paola, Mike, Max and Max’s charming little brother Alex, who was eight going on twenty-eight. While the adults ate chicken alfredo (except for vegetarian Paola) and the kids downed chicken fingers and fries, Mike leaned over Max, pulled up his shirt, and deftly attached a bag of cream-colored stuff to the feeding tube coming from his son’s stomach, holding it aloft for the food to go in. At the front of the ballroom, Ilyce, svelte in a shimmering blue gown, was getting ready for her annual thank-you. (more…)

Gene Therapy for Canavan Disease: Max’s Story

December 19, 2012

Tags: gene therapy, Canavan disease

Max celebrated his 15th birthday on October 13, 2012. His best friend is his brother Alex, a future neuroscientist.
I’m thrilled about the encouraging gene therapy results just published in Science Translational Medicine from Paola Leone, PhD and R. Jude Samulski, PhD, and colleagues. “Long-term follow-up after gene therapy for Canavan Disease” updates a project that has its origins in the mid 1990s. Canavan disease is a brain disorder present from birth.

I’ve been following some of the kids who’ve had the gene therapy. One patient in particular – Max Randell – has been in my human genetics textbook since age three, his progress updated with each edition. (more…)

Max Graduates!

June 4, 2012

Tags: Canavan disease, gene therapy, Ricki Lewis, The Forever Fix: Gene Therapy and the Boy Who Saved It, Ilyce Randell, Max Randell, Canavan Research Illinois, Jewish genetic diseases

14-year-old Max Randell, who has Canavan disease, is graduating middle school!
Max Graduates!

Tomorrow night, Max Randell will graduate from middle school. He even passed a test on the U.S. Constitution. I don’t think I could do that.

Max has Canavan disease. And thanks to gene therapy, he’s here to celebrate.

Canavan disease is an inherited disease that strips the insulation from nerve cells in the brain. It destroys neural function, beginning at birth and likely before, and the child loses the battle by age 8 -- unless she or he has gene therapy, still experimental (as are all gene therapies). (more…)

instruction
Project to engage students in helping families with rare genetic diseases
Book Club Reader's Guide
Many challenging questions to stimulate thought and discussion.
Instructor's Guide
38 discussion questions to get students thinking and talking about gene therapy, including the science, ethical issues, and the drug approval process.
Narrative science
The Forever Fix is the uplifting true story of 8-year-old Corey Haas, who was cured of hereditary blindness just 4 days after gene therapy.
College Textbooks
A spectacularly-illustrated, clearly written human anatomy and physiology textbook, used in pre-health profession programs throughout the U.S.
A highly engaging, clearly written, beautifully illustrated introduction to the science of human genetics for the non-scientist. Now in its 10th edition.
Nonfiction
An ideal starting point for anyone who wants to know more about genes, DNA, genomes, and the genetic ties that bind us all.

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