For the first time in 35 years of writing and revising my textbook Human Genetics: Concepts and Applications, I'm happy to report good news – real progress, and not hype.
The strides in 2025 range from effective gene therapies and drugs for devastating neurological conditions to a novel new painkiller. Here's a closer look at the good news!
Gene Therapy for Sanfilippo Syndrome
DNA Science first covered the story of Eliza O'Neill in 2014, here, when the parents of four-year-old Eliza, a vivacious, active, and adorable little girl, learned that she had inherited the rare brain condition Sanfilippo syndrome type A.
Her dad Glenn O'Neill remembered:
"'We found out the terrible news today. For now, I want to focus on her wonderful personality and life every day. One of my goals is to keep her happy and smiling for as long as possible. I love her so much.'
To continue reading, go to DNA Science, where this post first appeared.