Drug intelligence / Profile preview

recombinant adeno-associated virus serotype 2 empty vector

Development stage
Unknown
Modality
Gene Therapies
Administration
Intravenous, Intramuscular, Subretinal, Intrathecal
01

Overview

Recombinant adeno-associated virus serotype 2 (rAAV2) empty vector is a viral vector derived from the AAV2 serotype that lacks a therapeutic transgene or genetic payload. In the context of gene therapy development and clinical trials, it is primarily used as a negative control to distinguish the effects of the viral capsid and the host's immune response from the effects of transgene expression. AAV2 is the most well-characterized AAV serotype, known for its ability to infect a wide range of cell types, including neurons, hepatocytes, and muscle cells, by binding to the heparan sulfate proteoglycan (HSPG) receptor and the adeno-associated virus receptor (AAVR). While empty capsids are often considered impurities in therapeutic AAV preparations, they are also studied for their immunological properties and have been investigated as 'decoys' to sequester neutralizing antibodies, potentially enhancing the transduction efficiency of transgene-carrying vectors.

Other names
rAAV2-nullrAAV-2-nullrAAV 2-nullAAV2 empty capsidAAV-2 empty capsidAAV 2 empty capsidAAV2-null vectorAAV-2-null vectorAAV 2-null vector
02

Targets

HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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