Last week’s hot topic on Cystic Fibrosis titled CF Gene Defect Corrected Using Molecular ‘Scissor’ Approach to Stem Cell Therapy in Early Study was written by Alisa Woods.
The article is focused on a new study that suggests a method for correcting and improving stem cells derived from people with cystic fibrosis (CF), one that could lead to new ways of treating CF using a patient’s own cells. The report, titled “Robust method for TALEN-edited correction of pF508del in patient-specific induced pluripotent stem cells,“ appeared Feb. 9, 2016, in the journal Stem Cell Research and Therapy.
The researchers, led by Maria Vicenta Camarasa of Caubet-Cimera Foundation, Hospital Joan March, in Spain, wanted to devise a method to genetically correct patient-derived stem cells. The investigators decided to correct the CFTR pF508del mutation in stem cells taken from CF patients, as this is the most frequent gene mutation that leads to CF, occurring in about two-thirds of all patients.