What question did this study set out to answer?

To develop engineered virus-like particles capable of efficiently delivering gene editing ribonucleoproteins in vivo while avoiding off-target genomic damage.

January 1, 2022CellOpen Access

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

View Full Paper

Ask AI

Bookmark

Authors

SBSamagya BanskotaBroad Institute ARAditya RaguramBroad Institute SSSusie SuhUniversity of California, Irvine

Discussion

Member takes

Implication

Preclinical study demonstrates efficient in vivo delivery of base editors via engineered virus-like particles in mice, highlighting a safer approach for therapeutic gene editing.

Key Points

To develop engineered virus-like particles capable of efficiently delivering gene editing ribonucleoproteins in vivo while avoiding off-target genomic damage.
Engineered fourth-generation virus-like particles (eVLPs) to optimize cargo packaging, release, and cellular localization of base editor and Cas9 ribonucleoproteins.
Modulated cellular tropism using diverse surface glycoproteins and tested delivery across primary mouse and human cell types.
Administered single in vivo injections of eVLPs in mouse models targeting liver Pcsk9 and a model of genetic blindness to quantify editing efficiency and off-target activity.
Fourth-generation eVLPs achieved 63% liver editing in mice following a single injection, resulting in a 78% reduction in serum Pcsk9 levels.
Single-dose administration partially restored visual function in a mouse model of genetic blindness.
Off-target editing was virtually undetected both in vitro and in vivo, demonstrating improved fidelity compared to adeno-associated virus or plasmid delivery.

Cite This Study

Banskota et al. (2022) studied this question.

synapsesocial.com/papers/69d97f082a25b240b7a3ca4e https://doi.org/10.1016/j.cell.2021.12.021

View Full Paper

Ask AI

Bookmark

Implication

Preclinical study demonstrates efficient in vivo delivery of base editors via engineered virus-like particles in mice, highlighting a safer approach for therapeutic gene editing.

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

Discussion

Member takes

Implication

Key Points

Cite This Study

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

Discussion

Member takes

Implication

Key Points

Cite This Study

Also Consider

Also Consider