Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII clinical trials

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II

This study is testing a new gene therapy for boys aged 3-22 months with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. This condition means the body can't break down certain sugars, leading to health problems. The therapy aims to help the body make the missing enzyme itself to improve symptoms.