Scot A. Wolfe

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 study shows that systemic delivery of CRISPR-Cas9 nickase can suppress oncogene-amplified cancer progression in vivo.

The paper develops a modular platform for synthesizing bio-orthogonal macromolecular conjugates (BMCs) to enable targeted delivery of macromolecular therapeutics with improved efficiency and specificity.

SORT LNPs enable efficient Cas9-mediated repair of a pathogenic allele in skeletal muscle in a mouse model of LGMDR7.

This study develops stabilized Cas-embedded base editors (sCE_CBEs) that enable efficient and accurate C-to-T editing with minimal off-target effects in hematopoietic stem and progenitor cells.

This study shows that gene editing of HSCs without ex vivo culture reduces genotoxicity while maintaining therapeutic efficacy for beta-hemoglobinopathy treatment.

This study identifies mutations to improve prime editing efficiency in low-dNTP environments by enhancing MMLV-reverse transcriptase and reducing SAMHD1 activity.

The paper develops ABSOLVE-seq to assess genome editing off-targets linked to genetic variants using primary cells and sequencing, enabling evaluation of human genetic diversity's impact on gene editing specificity.

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

This study identifies ASRA as a leptin receptor antagonist that stimulates appetite and contributes to obesity by counteracting leptin signaling.

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.

The paper develops CRISPRme, a tool that integrates genetic diversity data to assess off-target risks of gene editing, finding allele-specific off-target effects in clinical gRNAs.

The paper develops a protocol to generate floxed alleles in zebrafish for cell-specific knockout, demonstrating its utility in studying gene function.

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.

This study optimizes the nuclear localization signal (NLS) of Cas12a to enhance editing efficiency in human primary cells without compromising specificity.

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 develops a compact prime editor with reduced reverse transcriptase activity, showing efficient editing and enabling flexible split-PE design for in vivo delivery.

This study identifies ZNF410 as a novel repressor of fetal globin by targeting the NuRD complex, offering a new therapeutic target for beta-hemoglobinopathies.

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.

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