"What keeps me coming back to work every day is a desire to do better."
Where I see patients (1)
Selected research
-
Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.
Cell reports. Medicine
-
GFP-on mouse model for interrogation of in vivo gene editing.
Nature communications
-
Integrated single-nuclei and spatial transcriptomic profiling of human sacrococcygeal teratomas reveals heterogeneity in cellular composition and X-chromosome inactivation.
bioRxiv : the preprint server for biology
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
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
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
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.