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「Hum Gene Ther[Journal]」の検索結果

4190件中 121件~140件表示    検索結果をPubMedで見る PubMedで見る

Gene Delivery to Human Limbal Stem Cells using Viral Vectors.

Nonviral delivery of GRIM-19 gene inhibits tumor growth with reduced local and systemic complications.

Bridging the Clinical Gap for DNA-based Antibody Therapy through Translational Studies in Sheep.

Utility of self-destructing CRISPR/Cas constructs for targeted gene editing in the retina.

Preclinical Evaluation of Chimeric Antigen Receptor-Modified T Cells Specific to Epithelial Cell Adhesion Molecule for Treating Colorectal Cancer.

State-of-the-Art 2019 on Gene therapy for Phenylketonuria.

Genetic based approaches to inherited metabolic liver diseases.

Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III.

Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.

Gene therapy for Pompe disease: the time is now.

Generation and clinical application of gene modified autologous epidermal sheets in Netherton syndrome - lessons learned from a phase 1 trial.

Enhancement of MicroRNA-200c On Osteogenic Differentiation and Bone Regeneration by Targeting SOX2-mediated Wnt Signaling and KLF4.

Visualization of retinal morphology in rd12mice by scanning electron microscopy.

Development of a gene-therapy vector for <i>RDH12</i>-associated retinal dystrophy.

INSIGHTS INTO GENE THERAPY FOR UREA CYCLE DEFECTS BY MATHEMATICAL MODELLING.

An engineered galactosylceramidase construct improves AAV gene therapy for Krabbe disease in twitcher mice.

Hematopoietic stem cell-targeted neonatal gene therapy with a clinically applicable lentiviral vector corrects osteopetrosis in oc/oc mice.

A new era for rare genetic diseases.

In vivo Exon Replacement in Mouse Atp7b Gene by CRISPR/Cas9 System.

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