D. Rabuka

Discovery and engineering of AiEvo2, a novel Cas12a nuclease for human gene editing applications

This paper identifies and engineers a novel Cas12a nuclease (AiEvo2) with improved specificity and efficacy for human gene editing, including applications in Huntington's disease and CAR-T therapy.

Anti-CRISPR protein mediated degradation of Cas9 in human cells

This study shows AcrIIA1 induces degradation of SpyCas9 and SauCas9 in human cells, suggesting a mechanism to modulate Cas9 activity for safer gene editing.

μCas, a novel class of miniature type-V Cas12f nucleases with diverse PAM

The paper describes μCas, a new class of small Cas12f nucleases with broader PAM preferences, enabling more genomic editing possibilities.

Tandem-Cleavage Linkers Improve the In Vivo Stability and Tolerability of Antibody-Drug Conjugates

This paper develops tandem-cleavage linkers to improve ADC stability and tolerability, showing better hematopoietic safety in rat studies.

A novel HER2-targeted antibody-drug conjugate offers the possibility of clinical dosing at trastuzumab-equivalent exposure levels

This paper develops a novel HER2-targeted ADC, CAT-01-106, showing improved efficacy and tolerability compared to T-DM1 at equivalent payload dosing.

Systematic identification of regulators of antibody-drug conjugate toxicity using CRISPR-Cas9 screens

This study uses CRISPR-Cas9 screens to identify genes regulating antibody-drug conjugate (ADC) toxicity, revealing endolysosomal trafficking regulators and mechanisms of ADC resistance.