Article
FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome.
Gene therapy - 1 Sept 2026
Lacher Richard K, Henson Kari, Wathen Lindsay N, Jones Caitlin, Arnold Tiffany, Rice McKenzie R, Carles Heather M, White Angela R, Ramsuchit Elizabeth, Murrey Darren, Raig Rebecca, Fisher Austen, Bucher Kaitlin, Westerkamp Grace C, Fritz Adam L, Gollaway Brooke M, Piloto Sebastian, Dismuke David, Robinson J Elliott, Williams Michael T, Vorhees Charles V, De Silva Erandi K, Tiwari Durgesh, Erickson Craig A, Pedapati Ernest V, Gross Christina
Abstract excerpt
Fragile X Syndrome (FXS) is the most common inherited form of intellectual disability. It is caused by a trinucleotide expansion in the 5' UTR of the Fragile X messenger ribonucleoprotein 1 (FMR1) gene leading to loss of expression of Fragile X messenger ribonucleoprotein (FMRP). There is currently no cure for FXS. We developed an FMR1 gene therapy based on an adeno-associated viral vector designed with strong...
Read the complete abstract on PubMedTopics
- Animals
- Fragile X Messenger Ribonucleoprotein 1
- Fragile X Syndrome