Thalassemia Patients and Friends

Discussion Forums => Announcements => Topic started by: Sunny2701 on April 16, 2018, 04:11:41 PM



Title: CRISPR Therapeutics Plans to Treat First Patients for Beta Thalassemia in Europe
Post by: Sunny2701 on April 16, 2018, 04:11:41 PM
CRISPR Therapeutics Plans to Treat First Patients for Beta Thalassemia in Europe

http://www.pharmalive.com/crispr-therapeutics-plans-to-treat-first-patients-for-beta-thalassemia-in-europe/


In this trial, researchers will harvest stem cells from the patients and, using CRISPR gene-editing tools, repair the cells so they have increased levels of fetal hemoglobin. Once completed, they will be infused back into the patients’ bodies via stem cell transplant. If all goes according to plan, the cells will generate a healthy supply of hemoglobin.

It will be the first human disease treated using CRISPR in Europe. China has several ongoing trials for various diseases using CRISPR. Salveen Richter, an analyst with Goldman Sachs, told the Telegraph, “As of the end of February 2018 there were nine registered clinical studies testing CRISPR-edited cells to treat various cancers and HIV infection in China.” In the U.S., there is only one. That is at the University of Pennsylvania for melanoma, sarcoma, and multiple myeloma.

“Certainly, 2018 promises to be the big year for clinical trials using CRISPR based genome editing,” Helen O’Neill, a geneticist from University College London (UCL), told the Telegraph. “Results presented by [CRISPR Therapeutics] at a hematology meeting showed that the method dramatically increased fetal hemoglobin in beta thalassemia patients’ cells. The therapy successfully edited over 90 percent of blood stem cells removed form patients, which were retransfused.”