Envisioning the Future Evolution of In Vivo Hematopoietic Stem Cell Engineering & Gene Editing Treatments
Time: 8:30 am
day: Day Two
Details:
- Establishing in vivo HSC engineering approaches by leveraging validated biology from ex vivo therapeutics to eliminate patient and manufacturing burdens and expand treatable patient populations
- Combining large gene insertions and lineage-specific control in vivo to enable multicellular interventions to diversify treatment opportunities
- Evaluating in vivo HSC engineering opportunities in gene editing methods, including Cas9, base editing, prime editing, and targeted insertions, alone and in combination with insertions
