Erik J. Sontheimer

The paper develops fully modified SpyCas9 guide RNAs that maintain or enhance genome editing efficacy in vitro and in vivo.

This paper introduces a Prime Assembly method for inserting large DNA fragments into the genome, achieving efficient and precise insertions up to 11 kilobase pairs.

This paper demonstrates that a compact ABE corrects the PiZ mutation in AATD mouse models, reducing liver disease and improving lung function.

The paper enhances Nme2Cas9 and Nme2SmuCas9 nucleases and base editors for improved efficiency and vector compatibility through mutagenesis and deaminase linker optimization.

The paper identifies PRC1.6 as a partner of the HUSH complex in promoter-specific provirus silencing through chromatin colocalization and functional assays.

The paper introduces a modular CRISPR vector assembly system called Fragmid, enabling rapid creation and testing of CRISPR technologies through combinatorial design and pooled screening.

This paper engineers Nme2Cas9 base editors to improve activity and targeting scope, enabling correction of MECP2 mutations with minimal off-target effects.

This study demonstrates the use of protecting oligos to enhance crRNA stability and potency for in vivo genome editing via AAV co-delivery in mice.

This study removes a hexanucleotide repeat expansion in C9ORF72 using CRISPR/Cas9 in cell and animal models, reducing disease hallmarks.

This study develops a compact adenine base editor (Nme2-ABE) that can be delivered via a single AAV vector to efficiently edit mouse genomic loci and revert disease mutations in a tyrosinemia model.

The paper demonstrates that multiple CRISPR-Cas systems and anti-CRISPR proteins function in zebrafish embryos, enabling combinatorial genome editing and recording.

This paper develops single-AAV vectors for precise in vivo genome editing using Nme2Cas9 with HDR templates or multiple sgRNAs, showing efficacy in treating HT-I and MPS-I mouse models.

This study shows that 5' modifications to DNA repair templates enhance precision genome editing efficiency in multiple animal models and human cells.

This paper demonstrates that circular ssDNA donors improve HDR efficiency and reduce off-target integration compared to linear ssDNA donors in mammalian cells.

The paper demonstrates that NmeCas9, a Cas9 ortholog, exhibits high-fidelity genome editing in human cells with minimal off-target effects and multiple PAM compatibility.

This study introduces NmeCas9 as a new in vivo genome editing tool, demonstrating its effectiveness in targeting specific genes in mice using a single recombinant adeno-associated vector.

The paper identifies and characterizes two new anti-CRISPR protein families that inhibit type II-C Cas9 systems, showing potent inhibition of NmeCas9 and cross-species activity.

C-BERST uses dCas9-APEX2 to map proteomes near genomic loci, identifying telomere and centromere-associated proteins.