"What keeps me coming back to work every day is a desire to do better."
Where I see patients (1)
My work
Fetal therapy offers hope for treating genetic disorders
An experimental therapy helped a family that had already lost two babies to a rare disorder with critical guidance from Dr. MacKenzie.
Selected research
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Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cells.
bioRxiv : the preprint server for biology
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Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus.
Molecular therapy. Methods & clinical development
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Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Molecular therapy : the journal of the American Society of Gene Therapy
Clinical trials
International Registry of Patients With Alpha Thalassemia
Number of fetuses diagnosed with alpha thalassemia who survive to birth, compared to number of fetuses diagnosed with alpha thalassemia who have fetal demise or are terminated in utero. This is measured in number of fetuses alive ...
Recruiting
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
Adverse and serious adverse events including, but not limited to, death within 24 hours after the procedure, stillbirth, death prior to initial hospital discharge,increased response with antibody development above that expected wi...
Recruiting
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