Academic Publication AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
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AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
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Adeno-associated virus as a delivery vector for gene therapy of human diseases
AbstractAdeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in differe...
Modulating nuclear stiffness and envelope barrier facilitates AAV nuclear entry and reduces immunogenicity
Low efficiency and immunotoxicity limit rAAVs clinical use. Here, the authors show that encapsulating rapamycin in PF127 micelles modulates cellular biomechanics, enhancing AAV-mediated gene transf...
In vivo site-specific engineering to reprogram T cells
Stable and cell-specific transgene expression can be achieved through in vivo site-specific integration of large DNA payloads using a two-vector system of enveloped delivery vehicles and adeno-asso...
Datopotamab Deruxtecan Versus Chemotherapy in Previously Treated Inoperable/Metastatic Hormone Receptor–Positive Human Epidermal Growth Factor Receptor 2–Negative Breast Cancer: Primary Results From TROPION-Breast01
PURPOSE The global, phase 3, open-label, randomized TROPION-Breast01 study assessed the trophoblast cell surface antigen 2–directed antibody-drug conjugate datopotamab deruxtecan (Dato-...
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Yes, highly correlated activity was mapped. An entry titled 'AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial' discusses this: No description provided.
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Yes, highly correlated activity was mapped. An entry titled 'Modulating nuclear stiffness and envelope barrier facilitates AAV nuclear entry and reduces immunogenicity' discusses this: Low efficiency and immunotoxicity limit rAAVs clinical use. Here, the authors show that encapsulating rapamycin in PF127 micelles modulates cellula...
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