Skip to main content

Filter Search Results

Publication Year

Animal Models

Topic Area

Methods

Funding Mechanism

Division of Comparative Medicine

Collaborating ICs

Selected Grantee Publications

In Vivo Expansion of Gene-Targeted Hepatocytes Through Transient Inhibition of an Essential Gene

De Giorgi et al., Science Translational Medicine. 2025.

https://pubmed.ncbi.nlm.nih.gov/39937884

This study explores Repair Drive, a platform technology that selectively expands homology-directed repair for treating liver diseases in male and female mice. Through transient conditioning of the liver by knocking down an essential gene—fumarylacetoacetate hydrolase—and delivering an untraceable version of that essential gene with a therapeutic transgene, Repair Drive significantly increases the percentage of gene-targeted hepatocytes (liver cells) up to 25% without inducing toxicity or tumorigenesis after a 1-year follow-up. This also resulted in a fivefold increase in expression of human factor IX, a therapeutic transgene. Repair Drive offers a promising platform for precise, safe, and durable correction of liver-related genetic disorders and may expand the applicability of somatic cell genome editing in a broad range of liver diseases in humans. Supported by ORIP (U42OD035581, U42OD026645), NCI, NHLBI, and NIDDK.

Biocompatibility and Bone Regeneration by Shape Memory Polymer Scaffolds

Gasson et al., Journal of Biomedical Materials Research Part A. 2025.

https://pubmed.ncbi.nlm.nih.gov/39404147

This study evaluates the potential of shape memory polymer (SMP) scaffolds for bone tissue engineering, focusing on their biocompatibility and ability to support bone regeneration. Researchers first demonstrated biocompatibility of SMP scaffolds in 12-week-old male Wistar rats and confirmed cell adhesion, proliferation, and differentiation, while promoting bone regeneration in 6 ­month-old male New Zealand white rabbits with induced bone defects. These scaffolds combine mechanical strength with the capacity to enhance biological healing, making them a promising tool for orthopedic applications. These findings highlight the potential of SMPs as a versatile platform for tissue engineering applications, combining structural support with biocompatibility to enhance bone repair and healing outcomes. Supported by ORIP (T32OD011083).