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09. März 2021 in Blood Adv

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

C. Li
K.A. Goncalves
T. Raskó
A. Pande
S. Gil
Z. Liu
Z. Izsvák
T. Papayannopoulou
J.C. Davis
H.P. Kiem
A. Lieber

02. September 2020 in Mol Ther

Targeting CD33 in chemoresistant AML patient-derived xenografts by CAR-CIK cells modified with an improved SB transposon system

M.C. Rotiroti
C. Buracchi
S. Arcangeli
S. Galimberti
M.G. Valsecchi
V.M. Perriello
T. Rasko
G. Alberti
C.F. Magnani
C. Cappuzzello
F. Lundberg
A. Pande
G. Dastoli
M. Introna
M. Serafini
E. Biagi
Z. Izsvák
A. Biondi
S. Tettamanti

20. August 2020 in JCI Insight

Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements

H. Wang
A. Georgakopoulou
C. Li
Z. Liu
S. Gil
A. Bashyam
E. Yannaki
A. Anagnostopoulos
A. Pande
Z. Izsvák
T. Papayannopoulou
A. Lieber

18. März 2019 in Nucleic Acids Res

Alternative 3' UTRs direct localization of functionally diverse protein isoforms in neuronal compartments

C. Ciolli Mattioli
A. Rom
V. Franke
K. Imami
G. Arrey
M. Terne
A. Woehler
A. Akalin
I. Ulitsky
M. Chekulaeva