CLN1 CRISPR/Cas9 KO Plasmid (m): sc-422390
- Target species: mouse
- 20 µg of transfection-ready, purified plasmid DNA; Suitable for up to 20 transfections
- CLN1 CRISPR/Cas9 Knockout (KO) Plasmid (m) is a pool of plasmids, each encoding Cas9 nuclease and a target-specific 20 nt guide RNA (gRNA) designed for maximum knockout efficiency using sequences derived from the GeCKO v2 library
- gRNA sequences direct Cas9 to induce site-specific double-strand breaks (DSBs) in the CLN1 genomic locus, resulting in gene knockout through non-homologous end joining (NHEJ)
- The puromycin resistance and RFP genes are flanked by LoxP sites, enabling removal of selection markers via Cre recombinase (Cre Vector: sc-418923) after establishing stable knockout cell lines
Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
CLN1 CRISPR/Cas9 KO Plasmid (m) | sc-422390 | 20 µg | $397.00 |
Overview
Ppt1 encodes palmitoyl-protein thioesterase 1 (CLN1), a lysosomal depalmitoylating enzyme that removes thioester-linked fatty acyl groups from S-palmitoylated proteins, facilitating their turnover and trafficking. Through regulation of lysosomal proteostasis, membrane protein recycling, and lipid handling, CLN1 supports neuronal homeostasis and intersects with autophagy–lysosome pathways. Loss of CLN1 activity is linked to neuronal ceroid lipofuscinosis biology, characterized by impaired lysosomal degradation and accumulation of storage material in post-mitotic tissues. In mouse systems, Ppt1 perturbation provides a tractable model to study how disrupted depalmitoylation impacts synaptic maintenance, neuroinflammatory signaling, and lysosome-dependent quality control.
CLN1 CRISPR/Cas9 KO Plasmid (m) is a pool of plasmids designed for targeted disruption of the Ppt1 gene in mouse cell lines. Each plasmid co-expresses a unique single guide RNA (sgRNA) targeting a distinct site within the Ppt1 together with the Streptococcus pyogenes Cas9 nuclease. The plasmids also encode GFP, allowing fluorescent identification and enrichment of successfully transfected cells by fluorescence microscopy or flow cytometry.
The multi-guide design increases the likelihood of generating insertions or deletions (indels) that disrupt the Ppt1 open reading frame following Cas9-mediated double-strand break formation. DNA breaks introduced by the CRISPR/Cas9 system are repaired through endogenous non-homologous end joining (NHEJ) pathways, frequently resulting in frameshift mutations that abolish CLN1 protein expression.
This CRISPR knockout system enables efficient generation of Ppt1-deficient cell models for investigation of CLN1 signaling, functional genomics studies, cancer biology research, and evaluation of therapeutic responses in human cell lines.
Key Features
- sgRNAs targeting Ppt1 exon(s) critical for CLN1 function
- Co-expression of SpCas9 and sgRNA from a single plasmid for simplified delivery
- GFP reporter for identification of transfected cells
- Pool of plasmids targeting multiple Ppt1 genomic sites to improve knockout efficiency
- Compatible with delivery by transfection
Design Variants
CRISPRs +/- HDRs
- gRNAs encoded by CLN1 CRISPR/Cas9 KO Plasmid (m) and CLN1 CRISPR/Cas9 KO Plasmid (m2) target distinct sites within the Ppt1 locus. One or both targeting designs may be available. See Related Products for availability.
- HDR donor constructs encoded by CLN1 HDR Plasmid (m) and CLN1 HDR Plasmid (m2) contain a puromycin resistance cassette and an RFP reporter flanked by Ppt1 homology arms to support homology-directed repair at defined Ppt1 target sites corresponding to the CRISPR/Cas9 KO designs. HDR donor availability may vary. See Related Products for availability.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.