OnTarget

OnTarget designs MiniPromoters by integrating user-provided genomic and cell-type-specific evidence, including accessible chromatin regions and histone modifications, with internal datasets to identify candidate promoters and enhancers for targeted gene expression in specific cells and tissues.

Key Features:

• MiniPromoter design: Generates compact DNA MiniPromoters capable of driving gene expression in specific cells and tissues.
• Genomic targeting input: Accepts specification of a gene of interest or custom genomic coordinates for promoter and enhancer identification.
• Cell-type-specific evidence: Incorporates accessible chromatin regions and histone modification data as cell-type-specific genomic evidence.
• Data integration: Integrates user-provided data with internal datasets to identify candidate promoters and enhancers tailored to cellular contexts.
• Disease-focused application: Has been applied to design MiniPromoters targeting cell populations relevant to Parkinson's Disease.

Scientific Applications:

• Gene therapy and precision medicine: Supports design of MiniPromoters to restrict therapeutic gene expression to target cell types, reducing off-target effects.
• Disease research (Parkinson's Disease): Enables creation of cell-population-specific regulatory sequences for studies and potential interventions in Parkinson's Disease.
• Custom therapeutic intervention design: Facilitates tailoring of promoter-enhancer combinations to specific cellular environments for therapeutic development.

Methodology:

Users provide genomic information and cell-type-specific evidence, which are combined with internal datasets to identify potential promoter and enhancer regions and design MiniPromoters optimized for the specified cellular contexts.