First gene therapy for Tay-Sachs disease successfully given to two children
By Miguel Sena-Esteves,
The Conversation
| 02. 14. 2022
Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development.
Tay-Sachs is a severe neurological disease caused by a deficiency in an enzyme called HexA. This enzyme breaks down a fatlike substance that normally exists in very small, harmless amounts in the brain. Without HexA, however, this fatlike substance can accumulate to toxic levels that damage and kill neurons.
One of the symptoms of this disease was first described in 1883 by British ophthalmologist Warren Tay, who saw a cherry-red spot on the back of the eye of affected infants. In 1887, American neurologist Bernard Sachs described the profound neurological symptoms of Tay-Sachs in a seminal paper:
“… Nothing abnormal was noticed until the age of two to three months, when the parents observed that the child was much more listless than children of that age. … The child would ordinarily lay upon its back, and was never able to change its position … it never attempted any voluntary movement … the child grew steadily weaker, it ceased to take its food...
Related Articles
By Diaa Hadid and Shweta Desai, NPR | 01.29.2026
MUMBRA, India — The afternoon sun shines on the woman in a commuter-town café, highlighting her almond-shaped eyes and pale skin, a look often sought after by couples who need an egg to have a baby.
"I have good eggs,"...
By George Janes, BioNews | 01.12.2026
A heart attack patient has become the first person to be treated in a clinical trial of an experimental gene therapy, which aims to strengthen blood vessels after coronary bypass surgery.
Coronary artery bypass surgery is performed to treat...
By Staff, ScienceDaily | 01.05.2026
Scientists at UNSW Sydney have developed a new form of CRISPR technology that could make gene therapy safer while also resolving a decades-long debate about how genes are switched off. The research shows that small chemical markers attached to DNA
...
Following a long-standing CGS tradition, we present a selection of our favorite Biopolitical Times posts of the past year.
In 2025, we published up to four posts every month, written by 12 authors (staff, consultants and allies), some in collaboration and one simply credited to CGS.
These titles are presented in chronological order, except for three In Memoriam notices, which follow. Many more posts that are worth your time can be found in the archive. Scroll down and “VIEW...
