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「Hum Gene Ther[Journal]」の検索結果

4188件中 141件~160件表示    検索結果をPubMedで見る PubMedで見る

Development of a Molecularly Stable Gene Therapy Vector for the Treatment of RPGR-associated X-linked Retinitis Pigmentosa.

In vitro validation of a CRISPR-mediated CFTR correction strategy for preclinical translation in pigs.

Treatment of CBS deficiency in mice using a minicircle-based naked DNA vector.

High-throughput quantification of in vivo AAV transduction with barcoded non-coding RNAs.

Multiplex Epigenome Editing of DRG Neuron Receptors Abolishes Redundant IL-6, TNF-α, and IL-1β Signaling by the Degenerative IVD.

Assessing the impact of Cyclosporine A on lentiviral transduction and preservation of human hematopoietic stem cells in clinically relevant ex-vivo gene therapy settings.

Retroviral UNC13D gene transfer restores cytotoxic activity of T cells derived from familial hemophagocytic lymphohistiocytosis type 3 patients in vitro.

An improved AAV vector for neurological correction of the mouse model of Mucopolysaccharidosis IIIA.

New frontiers of corneal gene therapy.

Safe and sustained expression of human iduronidase after adeno-associated virus 9 intrathecal administration in infant rhesus monkeys.

Constraints on human CD34+ cell fate due to lentiviral vectors can be relieved by valproic acid.

New Horizons for Immune Gene Therapy.

MALAT1-driven inhibition of Wnt signal impedes proliferation and inflammation in fibroblast-like synoviocytes through CTNNB1 promoter methylation in rheumatoid arthritis.

The alpha-1-antitrypsin promoter improves the efficacy of an AAV vector for the treatment of MNGIE.

A BICISTRONIC ADENOVIRAL VECTOR CARRYING CYTOSINE DEAMINASE AND GM-CSF INCREASES THE THERAPEUTIC EFFICACY OF CANCER GENE THERAPY.

A cetuximab-mediated suicide system in CAR-modified hematopoietic stem cells for cancer therapy.

Active immunoprophylaxis and vaccine augmentations mediated by a novel plasmid DNA formulation.

Enhanced transduction of Macaca fascicularis hematopoietic cells with chimeric lentiviral vectors.

Therapeutic delivery of miR-143 targeting tumor metabolism in poorly differentiated thyroid cancer xenografts and efficacy evaluation using 18F-FDG microPET-CT.

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D (LGMD2D).

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