Science Communications

Showing 1–10 of 34 posts

Science Communications MGX Presentation ISTH July 2026
07.28.26

A durable gene editing approach for Hemophilia A that enables curative levels of FVIII activity in non-human primates
Science Communications MGX Presentation ISTH July 2026
07.28.26

Stable expression of ATIII in non-human primates by CRISPR-mediated gene knock-in for the treatment of inherited thrombophilias
Science Communications MGX Presentation Keystone March 2026
03.20.26

CRISPR/Cas-Mediated APOС3 Knockout as a One-Time Treatment for Severe Hypertriglyceridemia
Science Communications MGX Presentation Keystone March 2026
03.13.26

Comprehensive Preclinical Specificity Profiling of MGX-001
Science Communications MGX Presentation ASGCT May 13 2025
05.13.25

Site-specific Integration of Therapeutic Transgenes with a Type V-K CAST System Engineered For Efficient And Targeted Human Genome Editing
Science Communications MGX Presentation ASGCT May 13 2025
05.13.25

In Vivo Genome Editing with an Ultra-Compact Type V Nuclease for All-In-One AAV Delivery
Science Communications MGX Presentation ASGCT May 13 2025
05.13.25

A Compact and Potent Type II CRISPR System for CNS Gene Knockdown via AAV Delivery
Science Communications Nature Communications
03.13.25

Integration of therapeutic cargo into the human genome with programmable type V-K CAST
Science Communications Nature Communications
01.13.25

DNA targeting by compact Cas9d and its resurrected ancestor
Science Communications MGX Presentation ASH Dec 9 2024
12.09.24

Site-Specific Insertion of a Factor VIII Gene Results in Durable Factor VIII Expression in Nonhuman Primates