Internal Reference: 2025-064

Market Need聽

The field of gene-targeted therapies has been revolutionized over the past decade with the introduction of CRISPR technology and clinical approval of one CRISPR-based therapeutic (Casgevy for treatment of sickle cell disease and transfusion-dependent 尾-thalassemia). Despite this success, modifications to the guide RNA (crRNA), or gRNA, are crucial for improving CRISPR-based gene therapies by enhancing specificity, reducing off-target effects, and increasing editing efficiency. However, complex nucleic acid-protein binding interactions within the CRISPR-Cas system make it difficult to fully modify the crRNA without negative impacting gene editing.

Technology Overview

九色视频 has introduced non-natural linkers into the crRNA of the CRISPR-Cas12a system that prevent exonuclease degradation, increase stability, and increase or maintain high editing efficiency while lowering toxicity. Non-natural linkers include carbon-based linkers and polyethylene glycol (PEG)-based linkers at select positions without compromising gene editing efficiency. The incorporation of the non-natural linkers can be readily done through standard solid phase oligonucleotide synthesis and can be added as an extra monomer, extending or altering the structure.

Commercial Advantages

• Enhanced nuclease stability and maintained activity in cells
• Rapid synthesis with commercially available monomers
• Precise and efficient genome editing tool
• Minimized use of nucleotide analogues with potential toxicity

Additional Information

• Researcher: Masad Damha聽
• 笔补迟别苍迟蝉:听US Provisional (Filed)
• Keywords: Gene Therapy, Chemical Modifications