RPI ID:
2022-044-301

Innovation Summary:
This gene editing system introduces a recombinant enzyme composed of an HIV integrase (HIV IN) tetramer and two or more Transcription Activator-Like Effectors (TALEs) bound via polypeptide linkers. The enzyme is designed to enable site-specific DNA insertion in eukaryotic cells with minimal off-target effects. The TALEs guide the construct to precise genomic loci, while the HIV IN facilitates integration of polynucleotides. The construct may be delivered via lentiviral capsids or vectors for in vivo applications.

Challenges / Opportunities:
Current genome editing tools like CRISPR-Cas systems suffer from off-target mutations and limited in vivo applicability. Recombinases require pre-inserted target sequences, reducing their flexibility. This technology addresses the need for accurate, site-specific editing without prior genomic modification. It opens opportunities for safer and more effective gene therapies, especially for genetic disorders requiring in vivo treatment.

Key Benefits / Advantages:
✔ High-accuracy DNA insertion with minimal off-target effects
✔ In vivo delivery via lentiviral vectors
✔ TALE-guided localization to specific genomic regions
✔ Configurable linker domains for structural optimization
✔ Potential for clinical gene therapy applications

Applications:
• In vivo gene therapy for genetic disorders

Keywords:
#geneediting #TALE #HIVintegrase #lentiviralvector #genetherapy #biotechnology

Intellectual Property:
PCT/US2024/026762 – Published, filed 29-Apr-2024