KIAA0319L Knockout Cell Line (HEK293)

The choice depends on whether you are studying KIAA0319L (AAVR, AAV receptor)'s role as the universal AAV cellular entry receptor — a recently characterized critical host factor for adeno-associated virus (AAV) gene therapy vectors. The Knockout line is the gold-standard tool for asking whether AAVR is required for AAV transduction — Pillay et al. (Nature 2016) identified KIAA0319L through unbiased haploid genetic screen as the essential receptor for AAV2 and all tested AAV serotypes (AAV1-9), establishing AAVR as a universal AAV receptor. AAVR mediates rapid endocytosis from the plasma membrane and trafficking to the trans-Golgi network. Overexpression of AAVR variants is useful for studying serotype-specific PKD domain interactions. For AAV gene therapy research, the EDITGENE KIAA0319L Knockout in HEK293 is uniquely valuable — HEK293 is the principal cell line for AAV vector production and characterization, making this knockout the gold-standard genetic null background for AAV transduction studies. Rescue with wild-type, PKD-domain-truncated, or C-terminal-truncated AAVR enables comprehensive studies of AAVR-AAV interactions. The knockout is critical for testing AAVR-independent AAV variants (AAV4 and AAVrh32.33 have been shown to use alternate entry pathways), engineering AAV-resistant cells, and developing strategies to enhance AAV transduction in target tissues (e.g., inner ear hair cells, where mature OHCs show reduced AAVR expression).

Primary applications: • AAV transduction studies: comprehensive AAV serotype testing (AAV1-9, AAVrh10, and emerging engineered capsids) — most serotypes are AAVR-dependent; AAV4 and AAVrh32.33 use AAVR-independent entry. • AAV gene therapy vector development: critical null background for confirming AAVR-dependence of new AAV variants and validating AAVR-targeted entry enhancement strategies. • AAV-AAVR interaction studies: serotype-specific PKD domain mapping using PKD-truncated AAVR rescue. • AAV escape mutant identification: directed evolution screens for AAVR-independent AAV capsids using the AAVR-null background. EDITGENE recommends this model as the gold-standard genetic tool for AAV gene therapy host factor research and AAV vector engineering.

Yes. AAVR rescue experiments are uniquely powerful for AAV gene therapy research: • Construct design: use a codon-modified KIAA0319L sequence with a small intracellular C-terminal tag (FLAG, HA). AAVR has extracellular MANSC domain, five PKD (polycystic kidney disease) domains, single transmembrane span, and C-terminal trafficking signals — preserve all elements. • PKD-domain-specific rescue: PKD1, PKD2 (most important for AAV2 binding), PKD3, PKD4, PKD5 truncations enable serotype-specific binding studies — different AAV serotypes engage distinct PKD domains. • C-tail-truncated rescue: C-terminal trafficking signal mutations may disrupt trans-Golgi network trafficking required for AAV infection without affecting binding. • Surface localization validation: confirm plasma membrane localization by cell surface staining before AAV transduction assays. • Functional readout: rescue should restore AAV serotype-specific transduction (luciferase or GFP reporter) — most serotypes are AAVR-dependent; AAV4 and AAVrh32.33 may show AAVR-independent transduction. HEK293 transduces efficiently with lentivirus and is the standard AAV production cell line, making rescue line generation straightforward and directly relevant to AAV vector engineering.