AAV8-RK-hBBS10 increases retinal function and thickness in mouse model of Bardet-Biedl syndrome
Oct. 23, 2024
Mutations in the BBS10 gene are the second most common cause of Bardet-Biedl syndrome (BBS). Researchers from Meiragtx Ltd. aimed to optimize and identify an AAV vector carrying the human (h)BBS10 gene, obtaining sustained efficacy as well as good safety for clinical translation for the treatment of BBS.