"What keeps me coming back to work every day is a desire to do better."
Where I see patients (2)
Selected research
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Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy.
Science translational medicine
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In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.
bioRxiv : the preprint server for biology
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Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases.
American journal of medical genetics. Part C, Seminars in medical genetics
Clinical trials
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Prenatal presentation of symptoms (e.g. hydrops) appearing on fetal imaging such as ultrasound and ECHO.
Recruiting
More about this study
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
More about this studyMy 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.
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Tender wagging care
Our therapy dogs spread joy and smiles at the bedside and throughout the hospital.
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