Bio Saga Blog - A Chronicle of Life Sciences & Informatics

"Prime editing" is more precise and more efficient than CRISPR and could herald a new era of genetic manipulation. The ability to edit the genes of human beings by changing the code of life -- DNA -- has dramatically improved over the last decade. Current tools, like  CRISPR-Cas9 and  base editors , are extremely powerful but have traditionally suffered from a lack of precision, high error rate and limited scope, hampering their ability to treat human genetic disease. A breakthrough gene-editing tool, developed by a team of researchers at Harvard University and unveiled Monday, has the ability to make extremely precise DNA edits, ushering in a new era of genetic manipulation. "In many respects this first report is the beginning rather than the end of a longstanding aspiration ... to be able to make any DNA change in any position of a living cell or organism including, potentially, human patients with genetic diseases," says David Liu, a chemist at Harvard'...

https://youtu.be/TdBAHexVYzc

https://time.com/5642755/crispr-gene-editing-humans/?amp=true It’s only been seven years since scientists first learned how to precisely and reliably splice the human genome using a tool called CRISPR , making it possible to think about snipping out disease-causing mutations and actually cure, once and for all, genetic diseases ranging from sickle cell anemia to certain types of cancer and even blindness. Doctors are plunging ahead in search of ways to use the relatively new technology to start treating patients. In China last November, scientist Jiankui He stunned—and dismayed—the genetic community when he announced he had already used CRISPR, which many believe still hasn’t been proven either safe or effective in human patients, to permanently alter the genomes of twin girls to be immune to HIV infection. He’s experiment was criticized because he edited the twins’ cells when they were embryos, therefore ensuring that every one of their cells is now changed, including their ...

https://singularityhub.com/2019/05/02/crispr-used-in-human-trials-for-the-first-time-in-the-us/amp/ CRISPR  just hit another landmark. Last week, the University of Pennsylvania (UPenn) confirmed that they have treated two cancer patients using the gene editing darling married with another biomedical wizard,  CAR-T . For now, it’s too early to tell if the treatment proved beneficial in either patient, but the team hopes to release a first batch of results in a conference or journal “ at an appropriate time .” It marks CRISPR’s first debut onto the US clinical scene, and it’s a long time coming. As early as mid-2016 the team had already received approval from the FDA to move ahead with their concept. Yet they didn’t register the trial  until early 2018 , taking careful precautions to not royally mess up CRISPR’s introduction into the biomedical mainstream. This is in stark contrast to China, which, due to laxer clinical registration rules,  kicked off its ...