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Biblio
Author
Title
Type
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Year
]
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Scaramuzza, Samantha
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2023
Lidonnici MRosa
,
Scaramuzza S
,
Ferrari G
.
Gene therapy for Hemoglobinopathies.
Hum Gene Ther. 2023.
PubMed
Google Scholar
Brusson M
,
Chalumeau A
,
Martinucci P
,
Romano O
,
Felix T
,
Poletti V
,
Scaramuzza S
,
Ramadier S
,
Masson C
,
Ferrari G
, et al.
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Mol Ther Nucleic Acids. 2023;32:229-246.
PubMed
Google Scholar
2022
Hardouin G
,
Antoniou P
,
Martinucci P
,
Felix T
,
Manceau S
,
Joseph L
,
Masson C
,
Scaramuzza S
,
Ferrari G
,
Cavazzana M
, et al.
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation.
Blood. 2022.
PubMed
Google Scholar
2019
Marktel S
,
Scaramuzza S
,
Cicalese MPia
,
Giglio F
,
Galimberti S
,
Lidonnici MRosa
,
Calbi V
,
Assanelli A
,
Bernardo MEster
,
Rossi C
, et al.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.
Nat Med. 2019.
PubMed
Google Scholar
Ferrua F
,
Cicalese MPia
,
Galimberti S
,
Giannelli S
,
Dionisio F
,
Barzaghi F
,
Migliavacca M
,
Bernardo MEster
,
Calbi V
,
Assanelli AAngelo
, et al.
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
Lancet Haematol. 2019.
PubMed
Google Scholar
2015
Neri T
,
Muggeo S
,
Paulis M
,
Caldana MElena
,
Crisafulli L
,
Strina D
,
Focarelli MLuisa
,
Faggioli F
,
Recordati C
,
Scaramuzza S
, et al.
Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts.
Stem Cell Reports. 2015.
PubMed
Google Scholar