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MacKenzie Lab »  Research

The MacKenzie Lab

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.

Blood-Brain Barrier Development

Studying how and when this barrier forms to better understand the unique environment surrounding the developing fetal brain and spinal cord.

Sacrococcygeal Teratomas

Elucidating the underlying biology of sacrococcygeal teratomas (SCTs) and developing predictive biomarkers and targeted treatments.

Maternal-Fetal Tolerance

We are studying, in collaboration with the Gardner Lab, the role of AIRE-expressing cells in the maintenance and disruption of a healthy pregnancy.  We are investigating fetal immune development to understand the contribution of the fetal immune system to pregnancy complications and prenatal disease.

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).

RNA Therapies

Evaluating the safety, biodistribution, and efficacy of prenatal therapies with antisense oligonucleotide) (ASO).

Gene Editing for Alpha-Thalassemia

Investigating different gene, base and prime editing technologies as a prenatal treatment for genetic blood disorders such as alpha-thalassemia major and sickle cell disease.... Read More

Gene Therapy Vectors

Examining the safety and biodistribution of different vectors for gene therapy delivery in the prenatal period.

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.

Clinical Trials

Translating research discoveries into clinical tools to help patients. Read more and reach out regarding our active clinical trials.

Translational Research

Transforming basic science research into IND-enabling studies for the prenatal treatment of inherited diseases.

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