Chimeric antigen receptor (CAR) T cell (CAR-T) therapies present options for patients diagnosed with certain leukemias. Recent advances of the technology included a method to integrate the CAR into the T cell receptor alpha constant (TRAC) locus to take advantage of the endogenous promoter and regulatory elements for CAR expression. This method used adeno-associated viral (AAV) vectors based on AAV6 to deliver the donor template encoding the CAR construct.
View Article and Find Full Text PDFEnviron Microbiol Rep
October 2024
Covalent Organic Frameworks (COFs) exhibiting kagome () structures are promising crystalline porous materials with two distinct pores. However, there are no reliable synthetic methods to exclusively target the over the polymorphic square-lattice () structure. To address this, we introduce a linker design strategy featuring bulky functional groups, which through steric interactions can hinder the net formation, thereby leading to a structure.
View Article and Find Full Text PDFMol Ther Methods Clin Dev
June 2024
Gene therapies and associated technologies are transforming biomedical research and enabling novel therapeutic options for patients living with debilitating and incurable genetic disorders. The vector system based on recombinant adeno-associated viral vectors (AAVs) has shown great promise in recent clinical trials for genetic diseases of multiple organs, such as the liver and the nervous system. Despite recent successes toward the development of novel bioengineered AAV variants for improved transduction of primary human tissues and cells, vectors that can efficiently transduce human Schwann cells (hSCs) have yet to be identified.
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