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Research Outputs
MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT
Journal
Identifier:
E226465
ISSN:
2329-0501
Overview
Overview
Research Outputs (15)
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A cellular disease model toward gene therapy of TGM1-dependent lamellar ichthyosis
Academic Article
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
Academic Article
An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells
Academic Article
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
Academic Article
Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy
Academic Article
Dissecting the Pharmacodynamics and Pharmacokinetics of MSCs to Overcome Limitations in Their Clinical Translation
Academic Article
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
Academic Article
Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application
Academic Article
Interactions between Retroviruses and the Host Cell Genome
Academic Article
It's all about location: Targeting the right spot for Wiskott-Aldrich syndrome
Academic Article
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
Academic Article
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
Academic Article
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency
Academic Article
The Pharmacology of Gene and Cell Therapy
Academic Article
Transcriptionally regulated and nontoxic delivery of the hyperactive Sleeping Beauty Transposase
Academic Article
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