Boston Children’s Hospital Patents New CRISPR Editing System That May Halt Triple-Negative Breast Cancer

Boston Children’s Hospital Patents New CRISPR Editing System That May Halt Triple-Negative Breast Cancer

Boston Children’s Hospital researchers recently completed a preclinical study evidencing ability for a tumor-targeted CRISPR gene editing system (encapsulated in a nanolipogel and injected into the body) that could effectively and safely halt the growth of the triple-negative breast cancer. This proof-of-principle study was conducted in human tumor cells and in mice and points toward a possible genetic treatment for triple-negative breast cancer. 

The new patent-protected strategy is reported online this week in journal PNAS.

Triple-Negative Breast Cancer (TNBC)

Lacking estrogen, progesterone, and HER2 receptors, this condition accounts for 12% of all breast cancers. It occurs more frequently in women under age 50, in African American women, and in women carrying a BCA1 gene mutation. Surgery, chemotherapy, and radiotherapy are the few treatment options for this highly aggressive, frequently metastatic cancer, which is in urgent need of more effective targeted therapeutics.

The Study

Led by Peng Guo, PhD, and Marsha Moses, PhD, in the Vascular Biology Program at Boston Children’s, this study represents the first successful use of targeted CRISPR gene editing to halt growth of a TNBC tumor in vivo (via injection into live, tumor-bearing mice). This new system is non-toxic and utilizes antibodies to selectively recognize cancer cells while sparing normal tissues.

The research team demonstrated that the novel CRISPR system could target breast tumors and knock out a well-known breast-cancer-promoting gene, Lipocalin 2, with an editing efficiency of 81% in tumor tissue. The approach attenuated growth by 77% in the mouse model and showed no toxicity in normal tissues.

The Novel CRISPR System

This novel approach encapsulates the CRISPR editing system inside a soft “nanolipogel” made up of nontoxic fatty molecules and hydrogels. Antibodies attached to the gel’s surface then guide the CRISPR nanoparticles to the tumor site. The antibodies are designed to recognize and target ICAM-1, a molecule the Moses Lab identified in 2014 as being a novel drug target for triple-negative breast cancer.

The particles efficiently enter cells, fuse with the tumor cell membrane, and deliver CRISPR payloads directly inside the cell. Once inside the cell, the CRISPR system knocks out the Lipocalin 2, an oncogene that promotes breast tumor progression and metastasis. Experiments showed that loss of oncogene inhibited the cancer’s aggressiveness and tendency to migrate or metastasize. The treated mice showed no evidence of toxicity.

Moving Forward

The researchers believe that the CRISPR platform could be adapted to treat pediatric cancers as well, and could also deliver conventional drugs. The studies for further investigation continue.


  • National Institutes of Health
  • Breast Cancer Research Foundation
  • Boston Children’s Hospital

Lead Research/Investigator

Led by Peng Guo, PhD

Marsha Moses, PhD

Call to Action: Are you interested in learning more about this patented CRISPR system? Connect with the Boston Children’s Hospital investigators or contact TrialSiteNews for contact information.