Targeted Delivery Of CRISPR Into Bone Tumors

Scientists in Hong Kong have developed a method to carry the CRISPR/Cas9 gene editing system into bone tumors, thus enabling therapeutic gene editing.

AsianScientist (Nov. 16, 2017) – In a study published in Biomaterials, scientists in Hong Kong have developed a delivery system for the CRISPR/Cas9 gene editing system that allows them to genetically modify tumors to treat bone cancer.

CRISPR/Cas9 is a genome editing technology which holds tremendous promise for cancer treatment. However, a major bottleneck for achieving the therapeutic potential of CRISPR/Cas9 is the lack of an in vivo targeted delivery system.

In this study, researchers at Hong Kong Baptist University (HKBU) have developed a novel targeted delivery system for CRISPR/Cas9 to edit VEGFA, a gene involved in the sprouting of new blood vessels, in osteosarcoma.

Led by Professor Lyu Aiping, Dean of the School of Chinese Medicine of HKBU, and Professor Zhang Ge, Director of Technology Development Division, the team used a nucleotide-based polymer called an aptamer to deliver components of the CRISP/Cas9 gene editing system into osteosarcoma tumors.

“Osteosarcoma, a very common primary malignant bone tumor in children and adolescents, is mainly treated by surgery and chemotherapy but the five-year post-surgery survival rate is a mere five to 20 percent,” said Zhang. “Aptamers, which are single-stranded oligonucleotides and could specifically recognize target cells have been widely used for in vivo targeted delivery of therapeutics. VEGFA has been reported to be a novel therapeutic target for osteosarcoma.”

In experiments using a mouse model, the aptamer facilitated selective distribution of CRISPR/Cas9 in both primary osteosarcoma and lung metastasis, leading to effective in vivo VEGFA genome editing. This inhibited orthotopic osteosarcoma malignancy and lung metastasis, and also reduced the formation of new blood vessels surrounding the tumor with no detectable toxicity.

“The tumor-specific aptamers, when conjugated with PPC polymers encapsulating CRISPR/Cas9, may facilitate therapeutic genome editing in tumors,” said Lyu.



The article can be found at: Liang et al. (2017) Tumor Cell-targeted Delivery of CRISPR/Cas9 by Aptamer-functionalized Lipopolymer for Therapeutic Genome Editing of VEGFA in Osteosarcoma..

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Source: Hong Kong Baptist University; Photo: Shutterstock.
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