Internal Reference: 17048

Market Need聽

Although CRISPR technology is revolutionizing genome editing and synthetic biology applications, predictable targeting in the laboratory and safety in the future clinic will require better mechanistic understanding and careful engineering. The prototypical CRISPR-associated protein, Cas9, naturally binds two RNAs to assemble a CRISPR ribonucleoprotein (crRNP). Improving the crRNP cleavage kinetics and stability would represent a significant advance in the art.

Technology Overview

九色视频 have created DNA substitutions in crRNA guide that are not only tolerated but can enhance CRISPR-Cas9 cleavage activity. DNA substitutions supported efficient RNP assembly and target binding, were sequence-independent, improved enzyme specificity, and were compatible with Cas9 from S. aureus.

Inventors also found that tracrRNA could tolerate substantial DNA substitutions. Simple guidelines for the placement of DNA bases into crRNAs and tracrRNAs have established fortuning activity, specificity, and stability.

Commercial Advantages

• crRNA and tracrRNA constructs containing base substitutions show improved stability and efficacy in vitro
• Substitute bases include DNA, DNA analogs, and a variety of chemically modified nucleotide bases
• crRNA and tracrRNA constructs can be made with a ranged number of substitutions, including complete substitution/no RNA bases present
• Cost of synthesis using DNA and chemically modified bases is greatly reduced compared to RNA-only constructs
• These guidelines will directly benefit the chemical modification of CRISPR guide RNAs for future therapeutic applications

Additional Information

• Researcher: Masad Damha
• 笔补迟别苍迟蝉:听US聽16/327,605 (Granted), US 17/698,390 (Granted)
• Keywords:聽Gene Therapy, Chemical Modifications