Tagged With "Gene Therapy"

Ms. Stacy Pike-Langenfeld, cofounder of the Krabbe disease advocacy KrabbeConnect, gave birth to her daughter Makayla in 2001. Makayla “was perfect until she wasn’t”—Krabbe disease visited her at 3.5 months of age and took her away at the age of 2. “There are no words that capture the vileness of infantile Krabbe disease. Absolutely none,” said Ms. Pike-Langenfeld. 20 years after the tragedy, hope has finally emerged for Ms. Pike-Langenfeld: her home state, Minnesota, has started universal...

Earlier this month, the New York Times reported on a boy, Kendric Cromer, 12, who was making the most progress in treating his Sickle Cell Disease (SCD) with gene therapy. The article discusses his 9-month-long journey to curing his SCD. However, I was disappointed the article did not discuss the gene-editing techniques involved in SCD gene therapeutics, or really the biological mechanism behind SCD–both fascinating topics in biology. SCD is caused by a point mutation in the gene that codes...