Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy
The American Journal of Human Genetics
Divya Sinha*, Benjamin Steyer*, Pawan K. Shahi, Katherine P. Mueller, Rasa Valiauga, Kimberly L. Edwards, Cole Bacig, Stephanie S. Steltzer, Sandhya Srinivasan, Amr Abdeen, Evan Cory, Viswesh Periyasamy, Alireza Fotuhi Siahpirani, Edwin M. Stone, Budd A. Tucker, Sushmita Roy, Bikash R. Pattnaik, Krishanu Saha, David M. Gamm. 2020. [link]
Media Coverage: UW-Madison [link]
Tracking and Predicting Human Somatic Cell Reprogramming Using Nuclear Characteristics
Scarless Genome Editing of Human Pluripotent Stem Cells via Transient Puromycin Selection
Stem Cell Reports
Manufacturing Cell Therapies: The Paradigm Shift in Health Care of This Century
National Academy of Medicine Perspectives
We utilize quantitative and bioengineering methods to advance the next generation of cell and gene therapies.
CRISPR-Cas9, nanoparticle delivery
We develop new tools and insights into the editing of the human genome. Projects include understanding DNA repair and nanoscale assembly of nucleic acids and novel nonviral polymeric delivery agents around protein-based CRISPR systems. We are advancing two projects in the NIH Somatic Cell Genome Editing Consortium.
Cell therapy manufacturing
Cells are living drugs that can be difficult to generate, scale-up, and quality control. Projects include monitoring and controlling the heterogeneity during gene modification and scale-up of stem cells and T cell immunotherapies (e.g., CAR T cells). We benefit from collaborations with the national Center for Cell Manufacturing.
Biomaterials + gene editing to generate new cell models
We are using customized biomaterials and genome editing to generate new human cell-based models of inherited disorders. Projects include correcting mutations within diseased cells and generate isogenic organoids that recapitulate morphogenesis and pathology seen in patients. This project exploits close collaboration with biologists and clinicians at the Waisman Center.
Science and Technology Studies
We seek to understand the dynamic and heterogeneous processes by which novel bioengineered objects get embedded into law and policy. Outputs are designed to invoke reflection among practicing scientists on the social commitments behind their choices when engineering human cells, as well as to inform regulations, institutional obligations, and state policy. We leverage collaborations within Holtz STS Center, Bio+Society Collaboratory, and Forum on Regenerative Medicine.
meet our lab
Members come from several programs including biomedical engineering, biophysics, molecular biology and medicine.
Amritava Das, PhD
Chimeric Antigen Receptor (CAR) T cell Engineering
PhD Candidate - Biomedical Engineering
Natural killer cell editing
We are supported by: