Benjamin P. Kleinstiver

The paper shows statins and mevalonate pathway inhibition increase STMN2 and ATF3, promoting neuronal regeneration in neurodegenerative disease.

OptiPrime predicts prime editing outcomes with high accuracy, enabling efficient therapeutic applications in human and mouse cells.

This paper develops a customized CRISPR-Cas9 base editor to correct the ACTA2 R179H mutation in a mouse model of MSMDS, improving survival and rescuing vascular and neurological phenotypes.

The paper identifies that C-terminal deletions in MECP2 cause Rett syndrome by reducing MeCP2 levels due to a specific reading frame shift, and shows a gene-editing strategy to correct this.

This paper introduces Protein2PAM, a deep learning model that predicts CRISPR-Cas PAM specificity from protein sequences, enabling the design of Cas variants with tailored PAM recognition.

This paper develops a CRISPR base editing strategy to correct the FD-causing mutation in human cells and mice, achieving up to 70% on-target correction and functional restoration.

This study shows splice modulators like branaplam reduce HTT CAG repeat expansion by affecting PMS1 expression, suggesting PMS1 as a new therapeutic target for Huntington's disease.

The paper assesses the impact of CRISPR perturbations on vascular endothelial cell functions at CAD loci, identifying variants that significantly affect endothelial phenotypes.

This paper describes the optimization of base editing to correct the SMN2 exon 7 mutation in SMA, achieving high editing efficiency and increased SMN protein levels in vitro and in vivo.

This study evaluates PAM-flexible dCas9 variants for bacterial CRISPR activation and repression, finding trade-offs between PAM flexibility and repression efficiency.

The paper challenges the validity of AcrVIA1-7 as type VI-A CRISPR anti-CRISPR proteins due to inconsistencies in bioinformatics and experimental results.

This study demonstrates the use of SpG and SpRY CRISPR-Cas9 variants in zebrafish and C. elegans for genome editing, showing improved accessibility to previously uneditable sites.

This paper engineers type V-K CRISPR-associated transposases to improve DNA insertion purity by introducing the HELIX system, achieving 99.3% simple insertion product purity.

This paper optimizes AsCas12a for combinatorial genetic screens in human cells by improving on-target and off-target activity and enabling higher-order multiplexing.

This study demonstrates allele-specific CRISPR/Cas9 editing of the P23H mutation in Rho-P23H mice, reducing mutant allele expression and delaying photoreceptor degeneration.

This paper demonstrates CRISPR-Cpf1-based transcription factors for inducible and tunable regulation of human gene expression, enabling combinatorial gene activation.