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Welcome to the Lab

Our research is focused on utilizing artificial nucleases for efficient targeted genome editing.
Engineering programmable nucleases for the targeted cleavage of a single site within a vertebrate genome for gene therapy applications:

  • We are particularly interested in applying Cas9 and Cpf1 (Cas12a) systems for therapeutic genome editing. We have established collaborations with a number of laboratories to target different diseases (beta-hemoglobinopathies in CD34+ HSPCs (Nature Med & Blood); LGMD2i; LGMD2G [Nature paper]; CGD; NF1; HPS1)
  • We are engineering Cas9 systems with improved precision and targeting range - see Cas9-Cas9 Nature Communications and Cas9-ZFPs Nature Methods paper
  • We are creating artificial zinc finger proteins for targeted gene regulation as therapeutic reagents using design and B1H selection approaches.

We are developing improved Cas9 delivery systems with the Khvorova, Sontheimer and Watts labs as a component of the NIH Common Fund SCGE.
We are applying programmable Cas9 nucleases to understand the influence of chromatin architecture on transcriptional activity - 4D Nucleome project.
We are applying programmable Cas9 nucleases for nuclease-based erradication of latent HIV genome structure- HIV project.
We are developing artificial ZFPs for targeted upregulation of NF1 - Gilbert Family Foundation

Recent Publications and Reagents

Our 3xNLS-SpCas9 nuclease achieves higher levels of genome editing in CD34+ Hematopoietic Stem Cells (Addgene

We have developed a nuclease-based strategy for the precise correction of pathogenic microduplications (Nature paper)

We have developed a nuclease-based strategy for the efficient repair of aberrant splicing mutations (Blood paper)

The Cas9-Cas9 plasmids from our CRISPR-Cas9 Nature Communications paper are distributed through Addgene.

The Cas9-pDBD plasmids from our CRISPR-Cas9 Nature Methods paper are distributed through Addgene.

The dCas9-effector plasmids from our CRISPRe Development paper with the Maehr lab are distributed through Addgene.

The Omega-based B1H binding site selection system Is being distributed through Addgene.

Positions Available: We are actively recruiting PostDocs, Graduate Students and Technicians

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