BREAKING NEWS! MECP2 Duplication is Reversible!
In a Nature article published online today, November 25, 2015, it was revealed that researchers at the Baylor College of Medicine, led by Dr. Huda Y. Zoghbi, have reversed MECP2 Duplication Syndrome in adult symptomatic mice using antisense oligonucleotide (ASO) therapy. In this landmark letter to Nature, mice treated with an experimental ASO administered through the central nervous system experienced the reduction of MECP2 protein to normal levels. Many impressive results were observed. Several behavioral tests were conducted, and in all cases, symptoms of hypoactivity, anxiety, and abnormal social behavior were resolved. Additionally, the seizure activity of the mice and abnormal EEG discharges were abolished. Lastly, initial studies demonstrated that reducing the MECP2 protein levels to the correct amount also normalized the expression of the other genes controlled by the MECP2 protein. Excitingly, the ASO was also administered to lymphoblastoid cells from MECP2 Duplication Syndrome patients, and MECP2 mRNA levels were restored to the correct concentration in these cells. Read More.
So how do I get involved?
Check out the 401 Project!
In 2012, The 401 Project was established. The primary reason for starting this project was, based on a collaborative idea between Collene Wright and Peter Anderson, that if we could get each duplication family to contribute $401.00 then we could raise the $80,000 needed to start the reversal experiment.
They didn’t stop there. Once the funds were raised, they then went on to create a website – www.401project.com. Today, “Cure MDS: 401 Project” has become an official subsidiary of the Rett Syndrome Research Trust and will not stop until there is a cure for MECP2 Duplication Syndrome. Please check out the video of the kiddos on the home page of this website. If you would like to have your child included on this page, please contact Peter or Collene by email at email@example.com
Furthermore, if you have any questions about how you can help to raise money for research; these are the parents you should talk to. They have a lot of ideas and can get you on the right path to helping us find a treatment for this disease.