Publications by authors named "Ghezal Beliakoff"
Purpose: Intravitreal delivery of therapeutic transgenes to the retina via engineered viral vectors can provide sustained local concentrations of therapeutic proteins and thus potentially reduce the treatment burden and improve long-term vision outcomes for patients with neovascular (wet) age-related macular degeneration (AMD), diabetic macular edema (DME), and diabetic retinopathy.
Methods: We performed directed evolution in nonhuman primates (NHP) to invent an adeno-associated viral (AAV) variant (R100) with the capacity to cross vitreoretinal barriers and transduce all regions and layers of the retina following intravitreal injection. We then engineered 4D-150, an R100-based genetic medicine carrying 2 therapeutic transgenes: a codon-optimized sequence encoding aflibercept, a recombinant protein that inhibits VEGF-A, VEGF-B, and PlGF, and a microRNA sequence that inhibits expression of VEGF-C.
Article Synopsis
- Advances in single-cell and spatial technologies are enhancing our ability to analyze gene expression in clinical tissue samples, particularly in breast cancer.
- By integrating various gene expression profiling techniques, researchers investigated molecular differences within tumor regions and identified key biomarkers linked to cancer progression.
- The study highlights that while each technology offers valuable data individually, their combined use reveals deeper insights into cancer heterogeneity, potentially influencing future research and treatment strategies in oncology.