Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

Author:

Mamcarz Ewelina1,Zhou Sheng1,Lockey Timothy1,Abdelsamed Hossam1,Cross Shane J.1,Kang Guolian1,Ma Zhijun1,Condori Jose1,Dowdy Jola1,Triplett Brandon1,Li Chen1,Maron Gabriela1,Aldave Becerra Juan C.1,Church Joseph A.1,Dokmeci Elif1,Love James T.1,da Matta Ain Ana C.1,van der Watt Hedi1,Tang Xing1,Janssen William1,Ryu Byoung Y.1,De Ravin Suk See1,Weiss Mitchell J.1,Youngblood Benjamin1,Long-Boyle Janel R.1,Gottschalk Stephen1,Meagher Michael M.1,Malech Harry L.1,Puck Jennifer M.1,Cowan Morton J.1,Sorrentino Brian P.1

Affiliation:

1. From the Departments of Bone Marrow Transplantation and Cellular Therapy (E.M., B.T., W.J., S.G.), Hematology (S.Z., Z.M., J.C., J.D., X.T., B.Y.R., M.J.W., B.P.S.), Therapeutics Production and Quality (T.L., M.M.M.), Immunology (H.A., B.Y.), Pharmaceutical Sciences (S.J.C.), Biostatistics (G.K., C.L.), and Infectious Diseases (G.M.), St. Jude Children’s Research Hospital, Memphis, TN; the Allergy and Clinical Immunology Division, Hospital Nacional Edgardo Rebagliati Martins, Lima, Peru (J.C.A.B.); the...

Funder

California Institute of Regenerative Medicine

National Heart, Lung, and Blood Institute

Assisi Foundation of Memphis

Division of Intramural Research, National Institute of Allergy and Infectious Diseases

American Lebanese Syrian Associated Charities

Publisher

Massachusetts Medical Society

Subject

General Medicine

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