Gene Editing: In Vivo In Waiting
This article was originally published in Start Up
Gene editing technologies could pose a challenge to gene therapy, bringing new capabilities such as correction of genes or insertion of novel genes at precise genome locations. Various technical barriers remain to maximizing the technique's efficiency, but long-term in vivo applications could transform treatment of a wide range of diseases.
You may also be interested in...
Biomedical researchers are rapidly adopting the new gene-editing technology CRISPR/Cas9 for lab experiments, but can CRISPR also become the basis for new treatments of human disease? At least two venture-backed start-ups can't wait to find out.
Gene editing has made a great many headlines of late, and one could be forgiven for thinking the field was saturated, but Homology Medicines Inc. offers something new. Its platform relies on a different mechanism, known as homologous recombination, and the start-up's management team believes the approach can get around some of the potential limitations found in other gene-editing technologies.
X4 Pharmaceuticals Inc. spun out from Sanofi to develop compounds that target the CXCR4/CXCL12 receptor pathway to restore immune cell surveillance against cancer.