Coenraads: In your opinion what are the elements that have made this consortium “work”?
Greenberg: Trust and openness, a willingness on the part of all three Principal Investigators to talk through any potential problems immediately as they come up. A willingness to check egos at the door so that we can work together for something that is more important than our individual advancement. Importantly the participants, Mandel, Bird, Greenberg and Coenraads like and trust each other.
Bird: We all have different backgrounds and interests, but we share a commitment to understanding Rett Syndrome. We compliment each other surprisingly well.
Mandel: The regular meetings and exchanges and the quality of the scientists involved have been key factors as well as the availability of sufficient funding for each of us to follow our scientific noses.
Coenraads: Fortunately science is not linear. There are technologies available now that weren’t available when the Consortium started. How does this impact your Rett research?
Greenberg: There are a lot of new technologies available – in particular Cre lines that will allow us to study the effect of MeCP2 loss in a relatively homogeneous population of neurons, CRISPR and Talen technology that will facilitate gene correction, and genomic technologies that are providing a new understanding of the role of methylation in the control of neuronal gene expression. Also, better equipment, such as microscopy will help.
Bird: The technologies for genetic modification have existed for a decade, but the advent of CRISPR has made this facile. Being able to edit genetic mistakes in patients is no longer a science fiction dream, but has become a real possibility. Exploring this option will be an important focus for the Consortium.
Coenraads: Harrison Gabel from Mike’s lab recently shared with me in an email: Our group meetings are essential to critically assessing our work. Each lab group has its own “world view,” and having that view shaken up every six months is very constructive. So I look forward to lots more critical assessments and worldviews getting shaken as together we get to the bottom of what MeCP2 does.
* Due to the success of the MECP2 Consortium, and its positive gene therapy findings, RSRT has just announced funding for a second consortium: the MECP2 Gene Therapy Consortium. Read more about this newly formed second collaboration.