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Clinical perspective on pluripotent stem cells derived cell therapies for the treatment of neurodegenerative diseases.
Adv Drug Deliv Rev
January 2025
Neurodegenerative Diseases Department, Kadimastem Ltd, Pinchas Sapir 7, Weizmann Science Park, Ness-Ziona, Israel; Department of Molecular Genetics, Weizmann Institute of Science, 76100, Rehovot, Israel.
Self-renewal capacity and potential to differentiate into almost any cell type of the human body makes pluripotent stem cells a valuable starting material for manufacturing of clinical grade cell therapies. Neurodegenerative diseases are characterized by gradual loss of structure or function of neurons, often leading to neuronal death. This results in gradual decline of cognitive, motor, and physiological functions due to the degeneration of the central nervous systems.
View Article and Find Full Text PDFOcular Safety and Toxicology of Subretinal Gene Therapy With rAAV.hPDE6A in Nonhuman Primates.
Transl Vis Sci Technol
January 2025
STZ eyetrial at the Centre for Ophthalmology, Tuebingen, Germany.
Purpose: Reports of gene therapy-associated retinal atrophies and inflammation have highlighted the importance of preclinical safety assessments of adeno-associated virus (AAV) vector systems. We evaluated in nonhuman primates (NHPs) the ocular safety and toxicology of a novel AAV gene therapy targeting retinitis pigmentosa caused by mutations in PDE6A, which has since been used in a phase I/II clinical trial (NCT04611503).
Methods: A total of 34 healthy cynomolgus animals (Macaca fascicularis) were treated with subretinal injections of rAAV.
Joint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes.
Stat Biopharm Res
March 2024
MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
In Phase I/II dose-finding trials, the objective is to find the Optimal Biological Dose (OBD), a dose that is both safe and shows sufficient activity that maximizes some optimality criterion based on safety and activity. In cancer, treatment is typically given over several cycles, complicating the identification of the OBD as both toxicity and activity outcomes may occur at any point throughout the follow up of multiple cycles. In this work we present and assess the Joint TITE-CRM, a model-based design for late onset toxicities and activity based on the well-known TITE-CRM.
View Article and Find Full Text PDFResponse to "Refinements in accelerated partial breast irradiation: Toward better efficacy and safety" by Yueqi Feng, Beina Hui, Ying Wang, Yongkai Lu.
Radiother Oncol
January 2025
Radiotherapy Centre, National Institute of Oncology, Budapest, Hungary; Department of Oncology, Semmelweis University, Budapest, Hungary.
Employing the Oncolytic Vesicular Stomatitis Virus in Cancer Virotherapy: Resistance and Clinical Considerations.
Viruses
December 2024
Thomas H. Gosnell School for Life Sciences, Rochester Institute of Technology, Rochester, NY 14623, USA.
Vesicular Stomatitis Virus (VSV) has emerged as a promising candidate for various clinical applications, including vaccine development, virus pseudotyping, and gene delivery. Its broad host range, ease of propagation, and lack of pre-existing immunity in humans make it ideal for therapeutic use. VSV's potential as an oncolytic virus has garnered attention; however, resistance to VSV-mediated oncolysis has been observed in some cell lines and tumor types, limiting its effectiveness.
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