Parts

Biological building blocks for gene therapy vector design. Browse by component type.

Delivery

Serotypes → AAV capsids for tissue targeting. Receptor binding, tropism, and transduction profiles for natural and engineered variants.
Backbones → Vector backbones with insert capacity constraints. ITRs, cloning sites, and selection markers.

Expression Control

Promoters → Transcriptional promoters for AAV and plasmid vectors. Expression strength and tissue specificity profiles.
RNA Pol III Promoters → Guide RNA promoters for CRISPR systems. U6, H1, and 7SK promoters for constitutive small RNA expression.
PolyA Signals → Transcription termination signals. Cleavage efficiency and mRNA stability effects.
Regulatory Elements → Post-transcriptional regulators, insulators, and enhancers. WPRE, cHS4, and related elements.

Editing Systems

Nucleases → CRISPR nucleases for genome editing. PAM sequences, cut types, and size for compact and standard variants.
Base Editors → Single-nucleotide conversion systems. CBE and ABE variants with editing windows and conversion types.
Prime Editors → Insertions, deletions, and all base substitutions without double-strand breaks. PE2–PE5 architectures.
Recombinases → Site-specific recombinases for conditional genetics, lineage tracing, and genomic integration.

Gene Regulation

Epigenome Editors → CRISPRa and CRISPRi systems. dCas9 fusions for transcriptional activation and repression.
Effector Domains → Modular transcriptional and epigenetic effectors. Activation, repression, and chromatin modification domains.

Payloads & Reporters

Proteins → Therapeutic proteins, enzymes, and transcription factors for gene therapy payloads.
Fluorescent Proteins → Fluorescent reporters for live and fixed imaging. Excitation, emission, brightness, and photostability profiles.
Bioluminescent Proteins → Luciferases for in vivo imaging and reporter assays. Substrate requirements and emission spectra.

Structural Elements

Localization Signals → Subcellular targeting sequences. NLS, signal peptides, mitochondrial targeting, and membrane anchors.
Linker Elements → 2A peptides, IRES sequences, and flexible linkers for polycistronic expression and fusion proteins.
Selection Markers → Antibiotic resistance markers for bacterial propagation and mammalian selection.