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MOLECULAR THERAPY (PRINT)
Rivista
Codice:
E112282
ISSN:
1525-0016
Dati Generali
Dati Generali
Pubblicazioni (57)
Pulisci
Ordina Pubblicazioni:
ascendente
decrescente
A Mouse Model of Gene Doping by AAV-IGF-1 Gene Transfer
Contributo in Atti di convegno
A novel vector for siRNA expression in mammalian cells that relies on polII-dependent transcription.
Articolo
AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy
Articolo
AAV-mediated Tyrosinase Gene Transfer Restores Melanogenesis and Retinal Function in a Model of Oculo-cutaneous Albinism Type I (OCA1).
Articolo
Adenoviral-mediated expression of Bone Morphogenetic Protein-4 in Mesenchymal Stem cells induces differentiation into the osteoblastic lineage and bone production in vivo.
Abstract
Amelioration of both functional and morphological abnormalities in the retina of a mouse model of ocular albinism following AAV-mediated gene transfer.
Articolo
Anti-Idiotypic CDR3 Vaccination Against Chronic B-Cell Lymphoma: DNA Vaccine Strategy
Abstract
Biochemical, Pathological, and Skeletal Improvement of Mucopolysaccharidosis VI After Gene Transfer to Liver but Not to Muscle.
Articolo
Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.
Articolo
Cationic PMMA Nanoparticles Bind and Deliver Antisense Oligoribonucleotides Allowing Restoration of Dystrophin Expression in the mdx Mouse
Articolo
Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse.
Articolo
Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene
Articolo
DNA Vaccine Strategy against chronic B-cell lymphoma: anti-idiotypic CDR3 vaccination
Abstract
Efficacy of a Combined Intracerebral and Systemic Gene Delivery Approach for the Treatment of a Severe Lysosomal Storage Disorder
Articolo
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA
Articolo
Eradication of Therapy-resistant Human Prostate Tumors Using an Ultrasound-guided Site-specific Cancer Terminator Virus Delivery Approach
Articolo
Exon skipping and Duchenne Muscular Dystrophy therapy: selection of the most active U1 snRNA-antisense able to induce dystrophin exon
Articolo
Extensive Methylation of Promoter Sequences Silences Lentiviral Transgene Expression During Stem Cell Differentiation In Vivo
Articolo
Extracellular superoxide dismutase is a growth regulatory mediator of tissue injury recovery.
Articolo
Fluoxetine ameliorates mucopolysaccharidosis type IIIA
Articolo
Fusion of HCV non-structural antigen to MHC Class II associated invariant chain enhances T cell responses induced by vectored vaccines in non-human primates.
Articolo
Gene Therapy Overcomes the Need for Multiple Infusions of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease
Articolo
Gene-mediated restoration of normal myofiber elasticity in dystrophic muscles.
Articolo
HIV-1 Mediated Insertional Activation of STAT5B and BACH2 Promotes the Formation of a Viral Reservoir in T Regulatory Cells
Articolo
Human inhalable antibody fragments neutralizing SARS-CoV-2 variants for COVID-19 therapy
Articolo
IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential.
Articolo
In vivo study of HIV-1 Tat arginine-rich motif unveils its transport properties
Articolo
In vivo study of HIV-1 Tat arginine-rich motif unveils its transport properties
Articolo
Inhibition of Receptor Signaling and of Glioblastoma-derived Tumor Growth by a Novel PDGFR beta Aptamer
Articolo
Inhibition of Receptor Signaling and of Glioblastoma-derived Tumor Growth by a Novel PDGFR? Aptamer.
Articolo
Lentiviral Vectors with a Reduced Splicing Interference Potential Have a Significantly Improved Safety Profile In Vivo
Articolo
Lentiviral vector mediated expression of human glucose 6-phosphate dehydrogenase in human hematopoietic stem cells
Abstract
Long-term Amelioration of Feline Mucopolysaccharidosis VI After AAV-mediated Liver Gene Transfer
Articolo
Long-term Amelioration of Feline Mucopolysaccharidosis VI After AAV-mediated Liver Gene Transfer
Articolo
Long-term Amelioration of Feline Mucopolysaccharidosis VI After AAV-mediated Liver Gene Transfer.
Articolo
Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector
Articolo
Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10mouse, a model of recessive retinitis pigmentosa.
Articolo
Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs
Articolo
Microglial vesicles improve post-stroke recovery by preventing immune cell senescence and favoring oligodendrogenesis
Articolo
Multi-lineage long-term expression of human glucose 6-phosphate dehydrogenase (G6PD) driven by the CMV promoter after lentiviral mediated transfer in primitive human hematopoietic cells (HSC) engrafting NOD/SCID mice.
Abstract
Multifunctional Aptamer-miRNA Conjugates for Targeted Cancer Therapy
Articolo
Multifunctional aptamer-miRNA conjugates for targeted cancer therapy.
Articolo
Nanoparticle-Mediated Delivery of Antisense Oligoribonucleotides Allows Restoration of Dystrophin Expression in the mdx Mouse
Articolo
Optimizing Gene Therapy for MPSVI: Overcoming Immune Responses and High Vector Doses
Articolo
Pharmacological Enhancement of alpha-Glucosidase by the Allosteric Chaperone N-acetylcysteine
Articolo
Preclinical PK and PD Studies on 2'-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model.
Articolo
Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome.
Articolo
Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model
Articolo
Proliferation of multiple cell types in the skeletal muscle tissue elicited by acute p21 suppression
Articolo
Short-interfering RNAs Induce Retinal Degeneration via TLR3 and IRF3
Articolo
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery
Articolo
Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration
Articolo
Targeting Axl With an High-affinity Inhibitory Aptamer
Articolo
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease
Articolo
Treatment with THI, an inhibitor of sphingosine-1-phosphate lyase, modulates glycosphingolipid metabolism and results therapeutically effective in models of disease
Articolo
VEGF-Induced Angiogenesis Does Not Have Favorable Effect on Muscle Perfusion: A Relevant Role of Log-Term Angiopoietin-1 Expression in the formation of Functional Blood Vessels
Abstract
dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice
Articolo
No Results Found
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