Denise A. Carbonaro-Sarracino

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Donald B. Kohn, Claire Booth, Kit L. Shaw et al. · New England Journal of Medicine · 2021 · 205 citations · Full text

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Bryanna Reinhardt, Omar Habib, Kit L. Shaw et al. · Blood · 2021 · 48 citations · Full text

Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Aaron Cooper, Georgia R. Lill, Kit L. Shaw et al. · Blood · 2017 · 30 citations · Full text

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

Denise A. Carbonaro-Sarracino, Alice F. Tarantal, C. Chang I. Lee et al. · Molecular Therapy — Methods & Clinical Development · 2019 · 20 citations · Full text

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Donald B. Kohn, Kit L. Shaw, Elizabeth Garabedian et al. · Blood · 2019 · 14 citations