frataxin protein

Jim Rusche from Repligen Corp. announced encouraging interim results from a phase 1 clinical trial of RG2833 in Friedreich's ataxia

posted on May 17, 2013 - 4:39pm
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the gene for the frataxin protein.

A gene therapy method tested in human cells ‘significantly’ increased production of frataxin, the protein deficient in Friedreich's ataxia

posted on August 8, 2012 - 6:00am
Human cells treated with engineered transcription activation-like effector (TALE) proteins produced two to three times more frataxin protein than did control cells, a team of researchers has reported.

A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake

posted on September 30, 2009 - 1:28pm
Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experimental compound being developed to treat this disease. MDA is supporting Repligen to develop this...