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  1. Pubblicazioni

MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT

Rivista
Codice:
E226465
ISSN:
2329-0501
  • Dati Generali

Dati Generali

Pubblicazioni (12)

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  • decrescente
A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB
Articolo
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
Articolo
Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy
Articolo
Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2
Articolo
Improvement of HSV-1 based amplicon vectors for a safe and long-lasting gene therapy in non-replicating cells
Articolo
In vivo generation of CAR T cells in the presence of human myeloid cells
Articolo
Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering
Articolo
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
Articolo
Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I
Articolo
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series
Articolo
Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo
Articolo
Therapeutic advantages of combined gene/cell therapy strategies in a murine model of {GM}2 gangliosidosis
Articolo
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