The Mali Lab
  • Home
  • Team
  • Research
  • Publications
  • Teaching
  • Join us!

Research

The major research thrusts in the laboratory are two-fold: one, development of molecular toolsets for genome, transcriptome, and proteome engineering and their application to systematic genome interpretation and gene therapy applications; and two, study and engineering of cell fate specification during development utilizing human pluripotent stem cells as the core model system. Given the parallels in phenotypes (such as self-renewal and tumor forming ability) between pluripotent stem cells and cancer cells, a key research thrust is also in dissecting aberrant cellular transformation processes such as during tumorigenesis.

Our research approach is curiosity-driven, integrating core expertise in genome engineering and stem cell engineering, with synthetic biology and materials science, and we are passionate about understanding and progressively engineering biology towards enabling gene & cell based human therapeutics. 
​
Selected publications:
  1. "Robust genome and cell engineering via in vitro and in situ circularized RNAs," Nature Biomedical Engineering (2025).
  2. "Charting and probing the activity of ADARs in human development and cell-fate specification," Nature Communications (2024).
  3. "Efficient in vitro and in vivo RNA editing via recruitment of endogenous ADARs using circular guide RNAs," Nature Biotechnology (2022).
  4. "Integrated genome and tissue engineering enables screening of cancer vulnerabilities in physiologically relevant perfusable ex vivo cultures," Biomaterials (2022).
  5. "Peptide tiling screens of cancer drivers reveal oncogenic protein domains and associated peptide inhibitors," Cell Systems (2021).​
  6. "Long-lasting analgesia via targeted in situ repression of Nav1.7 in mice," Science Translational Medicine (2021).
  7. "Defining the teratoma as a model for multi-lineage human development,"​ Cell (2020).
  8. "Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy," Nature Biomedical Engineering (2019).
  9. "In vivo RNA editing of point mutations via RNA-guided adenosine deaminases," Nature Methods (2019).
  10. "Mapping cellular reprogramming via pooled overexpression screens with paired fitness and single-cell sequencing readout," Cell Systems (2018).
  11. "Facile engineering of long-term culturable ex vivo vascularized tissues using biologically derived matrices," Advanced Healthcare Materials (2018).
  12. ​"Combinatorial CRISPR-Cas9 screens for de novo mapping of genetic interactions," Nature Methods (2017).
  13. "Rapidly evolving homing CRISPR barcodes," Nature Methods (2017).
  14. "Unraveling CRISPR-Cas9 genome engineering parameters via a library-on-library approach," Nature Methods (2015).
  15. "Cas9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering," Nature Biotechnology (2013).​
  16. “RNA-guided human genome engineering via Cas9,” Science (2013).
  17. “Barcoding cells using cell-surface programmable DNA binding domains,” Nature Methods (2013).
  18. “Site specific gene correction of a mutated beta-globin gene in human iPS cells derived from an adult patient with sickle cell disease,” Blood (2011).
  19. "Improved efficiency and pace of generating induced pluripotent stem cells from human adult and fetal fibroblasts," Stem Cells (2008).
  20. "Electrochemically programmed release of biomolecules and nanoparticles," NanoLetters (2006).
  21. "Facile fabrication of microfluidic systems using electron beam lithography," Lab on Chip (2006).
  22. "The dnaSET: A novel device for single-molecule DNA sequencing," IEEE Transactions on Electron Devices (2004).
Picture
We are grateful for the generous support of our funding sources:
Picture
Picture
Picture
Picture
Picture
Copyright Mali Lab @ UCSD. All rights reserved.