PC (protein C) is a plasma anticoagulant encoded by PROC; mutation in both PROC alleles results in neonatal purpura fulminans—a fatal systemic thrombotic disorder. In the present study, we aimed to ...

Adeno-associated viruses (AAVs) are promising delivery tools for gene therapy and are used in many clinical studies. However, less than ten AAV-based therapies have been approved to date. To mitigate ...

Existing gene therapies have primarily used a small number of naturally occurring AAV vectors limited by low delivery efficiency as well as problems with pre-existing immunity and manufacturability.

AMPA-type receptors (AMPARs) are rapidly inserted into synapses undergoing plasticity to increase synaptic transmission, but it is not fully understood if and how AMPAR-containing vesicles are ...

Chromatin-associated RNAs (caRNAs) form a relatively poorly recognized layer of the epigenome. The caRNAs reported to date are transcribed from the nuclear genome. Here, leveraging a recently ...

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