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  1. Pubblicazioni

MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT

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

Dati Generali

Pubblicazioni (15)

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A cellular disease model toward gene therapy of TGM1-dependent lamellar ichthyosis
Articolo
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
Articolo
An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells
Articolo
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
Articolo
Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy
Articolo
Dissecting the Pharmacodynamics and Pharmacokinetics of MSCs to Overcome Limitations in Their Clinical Translation
Articolo
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
Articolo
Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application
Articolo
Interactions between Retroviruses and the Host Cell Genome
Articolo
It's all about location: Targeting the right spot for Wiskott-Aldrich syndrome
Articolo
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
Articolo
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
Articolo
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency
Articolo
The Pharmacology of Gene and Cell Therapy
Articolo
Transcriptionally regulated and nontoxic delivery of the hyperactive Sleeping Beauty Transposase
Articolo
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