Research Awards
Fyodor Urnov, Ph.D. & Egor Prokhorchouk, Ph.D.
Sangamo BioSciences & Institute of Gene Biology, Moscow
Use of designed zinc finger protein nucleases in gene correction therapy for Rett Syndrome
$100,000
Lay Progress Report (August 2005)
Scientists at Sangamo are working to build proteins that would work as "genome editors" to directly correct the disease-causing mutation in RTT patients. Sixteen such proteins have been assembled; test-tube experiments have shown that one such protein is of very high quality, whereas other proteins require optimization that will be performed over the next 6-8 months. Scientists at the Centre for Bioengineering are creating a "humanized mouse" - an animal that carries the human MeCP2 gene disabled by the RTT mutation and that could be used to test the "genome editor" proteins.
Fyodor Urnov, Ph.D. & Egor Prokhorchouk, Ph.D.Sangamo BioSciences & Institute of Gene Biology, Moscow
Use of designed zinc finger protein nucleases in gene correction therapy for Rett Syndrome
$100,000
Lay Progress Report (August 2005)
Scientists at Sangamo are working to build proteins that would work as "genome editors" to directly correct the disease-causing mutation in RTT patients. Sixteen such proteins have been assembled; test-tube experiments have shown that one such protein is of very high quality, whereas other proteins require optimization that will be performed over the next 6-8 months. Scientists at the Centre for Bioengineering are creating a "humanized mouse" - an animal that carries the human MeCP2 gene disabled by the RTT mutation and that could be used to test the "genome editor" proteins.