Related Experiment Videos
Globin gene therapy: one (seemingly) small vector change, one giant leap in optimism
1National Human Genome Research Institute, National Institutes of Health, 49 Convent Drive, MSC 4442, Bethesda, Maryland 20892-4442, USA. tedyaz@nchgr.nih.gov
Summary
No abstract available in PubMed .
Related Concept Videos
Articles linked to this work by shared authors, journal, and citation graph.
Lhx2, a LIM homeobox gene, is required for eye, forebrain, and definitive erythrocyte development.
Development (Cambridge, England)·1997
Transduction of hematopoietic stem cells in humans and in nonhuman primates.
Stem cells (Dayton, Ohio)·1997
Induction of specific tolerance to MHC-disparate allografts through genetic engineering.
Experimental nephrology·1993
Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer.
The Journal of experimental medicine·1992
Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1.
Science (New York, N.Y.)·1992
Targeting the MNK1-MYH9 Axis Blocks YAP1 Recruitment to Prevent Thrombosis and Platelet Activation-Induced NETosis.
Molecular therapy : the journal of the American Society of Gene Therapy·2026
A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3.
Molecular therapy : the journal of the American Society of Gene Therapy·2026
A combinatorial EVs-miRNA signature mediates the anti-tumoral activity of NFAT3-regulated extracellular vesicles in aggressive cancers.
Molecular therapy : the journal of the American Society of Gene Therapy·2026
Triple AAV Intein-Mediated Gene Therapy Ameliorates Dystrophic Phenotype in MDC1A Mice.
Molecular therapy : the journal of the American Society of Gene Therapy·2026
Lineage-Tailored Vesicles from Human Retinal Ganglion-Like Cells Drive Metabolic Homeostasis and Bioenergetic Recovery in Glaucoma.
Molecular therapy : the journal of the American Society of Gene Therapy·2026
Dysregulated Proline Metabolism Contributes to Retinal Fibrosis in Neovascular AMD: Therapeutic Potential of Prolyl-4-Hydroxylase Inhibition.
Molecular therapy : the journal of the American Society of Gene Therapy·2026
Novel Intraoperative Augmented Reality-Guided Specimen Localization in Chest Wall Oncologic Surgery: A Case Report.
Journal of medical extended reality·2026
Synergistic SHH activation and BMP inhibition remodel progenitor niche for enhanced hair cell generation.
Science China. Life sciences·2026
Insulin gene-modified 3D stem cells for enhanced islet regeneration and systemic reversal of type 1 diabetes.
Stem cells translational medicine·2026
Ion-Driven Regulation of Ca2 + Flow by Ti/Zn Composite Activates ERK/MAPK Signaling and Maintains Mitochondrial Homeostasis to Promote Macrophage-Mediated Bone Regeneration.
Advanced science (Weinheim, Baden-Wurttemberg, Germany)·2026
Determinants of liver stiffness assessed by transient elastography in transfusion-dependent thalassemia.
Expert review of hematology·2026