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The Adeno-associated virus 214 (AAV214) receptor refers to the cell surface attachment and entry machinery utilized by the novel, engineered AAV214 capsid for cellular transduction. AAV214 is a synthetic capsid variant developed by Abeona Therapeutics, often identified from their AIM (AAV Inventory Management) library, and is designed for enhanced delivery of genetic material to the central nervous system (CNS) and ocular tissues. While most AAV serotypes utilize the Adeno-associated virus receptor (AAVR, also known as KIAA0319L) as a primary entry receptor, the specific attachment factors and co-receptors that define the unique tropism and superior transduction efficiency of AAV214 remain undefined in the public scientific literature. This machinery is a critical component of gene therapy platforms, such as AAV214-CLN3, which aims to treat neurodegenerative conditions like juvenile Batten disease by delivering functional genes to affected neurons and retinal cells. Understanding the molecular interactions between the AAV214 capsid and its host cell receptors is essential for optimizing tissue-specific targeting and minimizing off-target effects in clinical applications.
Viral vector-mediated gene transduction
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