Abstract: Provided herein are compositions, systems, and methods comprising effector proteins and uses thereof. These effector proteins may be characterized as CRISPR-associated (Cas) proteins. Various compositions, systems, and methods of the present disclosure may leverage the activities of these effector proteins for the modification, detection, and engineering of nucleic acids.

Abstract: Provided herein are compositions, systems, and methods comprising effector proteins and uses thereof. These effector proteins may be characterized as CRISPR-associated (Cas) proteins. Various compositions, systems, and methods of the present disclosure may leverage the activities of these effector proteins for the modification, detection, and engineering of nucleic acids, including editing target nucleic acids, as well as, the treatment of diseases and disorders associated with the dystrophin gene (DMD).

Abstract: Therapeutic methods and compositions for the in utero or postnatal treatment of diseases associated with alternative splicing are provided. Compositions of the disclosure include delivery nanoparticles with an inner region surrounded by a nucleic acid scaffolding that is, in turn, linked to therapeutic agents that promote healthy mRNA splicing phenotypes in fetal cells when the compositions are delivered to a fetus in utero or in a patient after birth.