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Nucleic Acid-Based Therapies
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Platform for AAV Delivery to Skin
Emerging startup: A platform technology from George Church's lab enables delivery of gene therapy via the skin. (Image credit: Carl Schoellhammer, Denitsa Milanova, Humberto Trevino, Cody Cleveland, Jeff Wyckoff, Anna Mandinova, George Church,…
DBD
- Grant Zimmermann
Investigators
- George Church
Evolution of Sequence-Defined Highly Functionalized Nucleic Acid Polymers
DESCRIPTION Template-directed synthesis of modified DNA sequences can provide a rich source of material for generating new therapies featuring modified nucleic acid polymers. The Liu lab has previously developed pioneering methods to generate…
DBD
- Irit Ben-Chelouche
Investigators
- David Liu
Enhancers of Gene Therapy
Currently available gene therapy technologies rely on either plasmid DNA or viral vector transduction to enable gene transfer. However, such methods often suffer from poor direct expression and insufficient incorporation of foreign genetic material.…
DBD
- Vivian Berlin
Investigators
- David Knipe
- Megan Orzalli
Inhibition of STAG1 using ASOs for treatment of STAG2-deficient cancers
The cohesin complex is one of the most frequently mutated protein complexes in cancer. Cohesin mutations are associated with poor survival and there are no therapies that selectively target cohesin-mutant cancers. STAG2 is the most frequently…
DBD
- Irit Ben-Chelouche
Investigators
- Karen Adelman