First CRISPR Trial to Edit DNA in Human Body
Biotech companies Allergan and Editas Medicine have announced that the Brilliance Phase 1/2 clinical trial of AGN-151587 (EDIT-101) is open for patient enrollment. The clinical trial will be the world’s first in vivo study of a CRISPR-based genome editing medicine, where the editing takes place inside the human body.
“Beginning patient enrollment in the AGN-151587 clinical trial with our partners at Editas is an important step toward our goal of developing a game-changing, transformative, CRISPR-based medicine,” said Allergen researcher David Nicholson.
AGN-151587 is an experimental medicine under development for the treatment of Leber congenital amaurosis 10 (LCA10), an inherited form of blindness caused by mutations in the CEP290 gene.
SingularityHub emphasizes that, while CRISPR-based medicine has been tested in humans, this is the first study to use CRISPR to edit DNA inside the human body.
The clinical trial patients will have the treatment, which was approved by the FDA in December, injected behind their retinas. The hope is that the patients’ DNA will repair itself in a way that restores normal protein function, ultimately fixing their photoreceptor cells and letting them see.
High-energy particle beams target tumors precisely. Researchers led by scientists at the University of Strathclyde have proposed a new way of concentrating radiotherapy doses in tumors, while minimizing damage to healthy cells. In a study published in Scientific Reports, the researchers suggest that focusing high-energy particle beams on a small spot deep inside the body could potentially enable clinicians to target cancerous tumors precisely, while reducing the dose to surrounding tissue.
Advances toward drugs able to remove aging cells. Researchers at University of Southern California have advanced toward an understanding of how the aging process works. In a study published in Journal of Biological Chemistry, the researchers show that aging senescent cells stop producing a class of chemicals called nucleotides, which are the building blocks of DNA. The research results could find applications in the emerging field of senolytics, the development of drugs that may be able to eliminate aging cells, and open the door to better cancer treatments and revolutionary new drugs for aging patients.
Laser-guided microrobots precisely deliver cancer drugs. Caltech researchers are working on an entirely new form of treatment: microrobots that can deliver drugs to specific spots inside the body while being monitored and controlled from outside the body. Once the microrobots, described in a research paper published in Science Robotics, arrive in the vicinity of a tumor, a near-infrared laser beam is used to activate them. Because the microrobots absorb the infrared light strongly, they briefly heat up. At that point, the microrobots' bubble jets activate, and the microrobots swarm and deliver their medication payload.
3D-printed pill profiles bacteria in the gut. Researchers led by Tufts University engineers have developed a 3D-printed pill, described in a study published in Advanced Intelligent Systems, which samples bacteria found in the gut, known as the microbiome, as it passes through the gastrointestinal tract (GI). The ability to profile bacterial species throughout the GI tract could have important implications for the diagnosis and treatment of diseases and conditions that are affected by the microbiome.
Tracking and killing cancer cells that escape surgery. Biomedical engineers at Vanderbilt University have discovered a method to track down and kill escaping cancer cells during tumor removal surgeries. In a research paper published in Science Advances, the scientists report that nanoparticles injected during and after tumor removal kill all cancer cells loosed as a result. The method, which uses the body's own defenses in the form of modified lipid nanoparticles adhered to white blood cells, could also help patients who already suffer from metastatic cancer.
Protein sensor finds damaged DNA and oversees repair. Scientists at University of Pittsburgh have advanced toward an understanding how one protein in particular keeps DNA damage in check. The protein, called UV-DDB, which stands for ultraviolet-damaged DNA-binding, is useful beyond safeguarding against the sun. New evidence, described in a study published in Nature Structural and Molecular Biology, points to UV-DDB being a scout for general DNA damage and an overseer of the molecular repair crew that fixes it.
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