Research
Our lab is focused on developing better ways to diagnose and treat genetic diseases before birth by leveraging novel fetal molecular therapies, understanding basic fetal biology, and conducting clinical trials.
Fetal Biology
The MacKenzie lab is dedicated to using both clinical and basic science tools and techniques to study fetal development to continue to develop new prenatal diagnostics.
The MacKenzie lab is dedicated to using both clinical and basic science tools and techniques to study fetal development to continue to develop new prenatal diagnostics.
Prenatal Molecular Therapies
Molecular therapies have emerged as a powerful platform for the treatment of many genetic disorders. Prenatal gene therapy/editing of somatic cells before disease onset can offer significant advantages over postnatal approaches and is especially important for diseases with high perinatal mortality. Our lab focuses on studying protein replacement therapies, gene therapy vectors (viral and non-viral) and technologies (ASOs, gene editing, gene replacement).
Molecular therapies have emerged as a powerful platform for the treatment of many genetic disorders. Prenatal gene therapy/editing of somatic cells before disease onset can offer significant advantages over postnatal approaches and is especially important for diseases with high perinatal mortality. Our lab focuses on studying protein replacement therapies, gene therapy vectors (viral and non-viral) and technologies (ASOs, gene editing, gene replacement).
Translation to the Clinic
We translate research discoveries into clinical tools to help patients through our ongoing translational science and active clinical trials. There are two active clinical trials of prenatal therapies tested in the MacKenzie lab including: In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases and In Utero Hematopoietic Stem Cell Transplantation for Alpha Thalassemia Major.
We translate research discoveries into clinical tools to help patients through our ongoing translational science and active clinical trials. There are two active clinical trials of prenatal therapies tested in the MacKenzie lab including: In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases and In Utero Hematopoietic Stem Cell Transplantation for Alpha Thalassemia Major.
