The Translational Institute for Molecular Therapeutics was formed in February and was the first team to deliver a human gene therapy for Tay-Sachs, and is now working with families on five other diseases, including one family fighting UBA5.
The Translational Institute for Molecular Therapeutics was formed in February and was the first team to deliver a human gene therapy for Tay-Sachs, and is now working with families on five other diseases, including one family fighting UBA5.
