This product consists of a CRISPR/Cas9-edited polyclonal knockout cell population derived from HEK293T cells, engineered for targeted disruption of the ATXN1L gene. The polyclonal format provides a heterogeneous pool of edited cells with diverse loss-of-function alleles, enabling robust population-level analysis of ATXN1L-dependent phenotypes. This model serves as a powerful tool for dissecting the molecular functions of ATXN1L in transcriptional regulation and its role in CIC repressor complex activity, without relying on clonal isolation. The CRISPR/Cas9-mediated gene disruption abolishes ATXN1L protein expression, creating a versatile system for studying downstream effects in a human cell context.
HEK293T cells are a widely utilized human embryonic kidney cell line that stably expresses the SV40 large T antigen. This modification permits episomal replication of plasmids containing the SV40 origin of replication, significantly boosting transient protein expression and lentiviral production efficiency. The ATXN1L knockout HEK293T polyclonal cells retain these advantageous features, allowing high-efficiency transfection and biochemical manipulation. Consequently, this model combines the genetic tractability of HEK293T cells with a loss-of-function background for ATXN1L, facilitating detailed mechanistic studies of this transcriptional regulator in a well-characterized cellular environment.
ATXN1L is a paralog of ATXN1 and functions as a key modulator of CIC-mediated transcriptional repression. It physically interacts with CIC (capicua) and corepressor complexes to transcriptionally regulate target genes, including members of the ETS family of transcription factors and other neuronal development genes. ATXN1L participates in Notch signaling networks, where it intersects with pathway components such as Notch receptors and RBPJ. Disruption of ATXN1L dysregulates CIC target gene expression, potentially altering transcriptional outputs critical for neuronal function. This molecular interplay positions ATXN1L at the intersection of transcriptional regulatory mechanisms relevant to spinocerebellar ataxia and broader neurodegenerative processes.
The ATXN1L knockout in the HEK293T background offers significant scientific value for investigating the gene??s role in CIC repressor complex dynamics and target gene regulation. Unlike neuronal cells, HEK293T provides a simplified yet biologically relevant platform to examine ATXN1L??s molecular interactions and transcriptional effects without the confounding factors of specialized neural environments. The model supports a range of biochemical and cell-based assays, enabling the direct assessment of ATXN1L??s contribution to CIC-dependent gene repression and its impact on Notch-associated transcriptional programs. This facilitates the elucidation of pathogenic mechanisms underlying SCA1 and related disorders.
This polyclonal knockout model is ideally suited for advanced research applications including functional characterization of ATXN1L in transcriptional regulation, investigation of spinocerebellar ataxia pathogenic mechanisms, screening for modifiers of CIC-ATXN1 complex activity, and drug target validation for neurological disorders. Compatible experimental techniques encompass Western blotting for ATXN1L protein levels, RT-qPCR for ETS target gene expression, co-immunoprecipitation of ATXN1L with CIC, reporter assays for CIC-responsive elements, and immunofluorescence for subcellular localization. These cells empower researchers to dissect ATXN1L biology with precision. For further details, please contact Ascent Research.
