I have the same mission as Benioff Children’s Hospital Oakland: to treat and improve the outcome for every child and family who comes here for care and support.
Where I see patients (1)
Selected research
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Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction.
Molecular therapy : the journal of the American Society of Gene Therapy
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Oligonucleotide Synthesis Errors Are a Source of Untoward Variation in HDR-Mediated Gene Editing.
Genes
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Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease.
The New England journal of medicine
Clinical trials
A Study of Participants with ?-Thalassemia Treated with Betibeglogene Autotemcel
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event [SAE]): - Any newly diagnosed malignancy - Neutrophil engraftment failure: defined as healthc...
Recruiting
More about this studyThe EndRAD Trial: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adole...
The primary objective of this study is the two Year Event-free Survival for patients with high-risk or recurrent B-ALL who proceed to HCT and who are NGS-MRD negative when treated with a non-TBI preparative regimen.
Recruiting
More about this studyContact me
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