In vivo gene editing tools for blood disorders may be delivered via lipid nanoparticles, according to researchers. The findings were published in a proof-of-concept paper in Science. This method of delivery could reduce costs and increase access to gene therapies.
The model was created by researchers at the Perelman School of Medicine at the University of Pennsylvania. By delivering gene editing tools in this way, diseased blood cells could be modified directly within the body.
The idea behind in vivo gene editing is to eliminate the need for conditioning regimens like radiation or chemotherapy, both of which are currently used to prepare patients with diseases like sickle cell disease and beta thalassemia for a hematopoietic cell transplant or ex vivo gene therapy.
“In our study, we are providing a cell-specific targeted lipid nanoparticle encapsulating mRNA therapeutics/editors as a platform technology that can be used for in vivo cellular reprogramming in many diseases in need of a precisely targeted gene therapy modality,” said senior author Hamideh Parhiz, PharmD, PhD. “Here, we combined the targeted platform with advances in mRNA therapeutics and RNA-based genomic editing tools to provide a new way of controlling hematopoietic stem cell fate and correcting genetic defects.”
“These findings may potentially transform gene therapy, not only by allowing cell-type specific gene modification in vivo with minimal risk, which could allow for previously impossible manipulations of blood stem cell physiology, but also by providing a platform that, if properly tuned, can correct many different monogenic disorders,” said Laura Breda, PhD, a research assistant professor with the Division of Hematology at Children's Hospital of Philadelphia. “Such novel delivery systems may help translate the promise of decades of concerted genetic and biomedical research to ablate a wide array of human diseases.”
Source: Children’s Hospital of Philadelphia, July 27, 2023.