C12N9/224

COMPOSITIONS AND METHODS FOR PRECISE EDITING OF HUMAN DYSTROPHIN

Disclosed herein are systems, compositions, and methods for modifying the human dystrophin gene (DMD). Systems, compositions, and methods may comprise a compact Type V CRISPR-associated (Cas) protein, an RNA-dependent DNA polymerase, and/or one or more guide nucleic acids or uses thereof. These systems, compositions, and methods may be useful for treating diseases such as Duchenne muscular dystrophy (DMD).

RECOMBINANT TYPE I-F3 TRANSPOSON-ASSOCIATED CRISPR-CAS SYSTEMS AND METHODS OF USE
20260110001 · 2026-04-23 ·

This invention relates to recombinant nucleic acid constructs encoding Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas systems and transposon 7-like (Tn7-like) transposon systems for DNA integration, as well as methods of using the same.