Rett Syndrome Research Trust Blog

• Treating Synaptic Dysfunction
  RETT SYNDROME RESEARCH TRUST WEBSITE Last month brought me to Houston, Texas to attend a fascinating meeting organized by Huda Zoghbi and Morgan Sheng and co-sponsored by RSRT. Entitled Disorders of Synaptic Dysfunction, the event was the inaugural symposium of the recently established Jan and Dan Duncan Neurological Research Institute, directed by Dr. Zoghbi. The [...]
  
  
• The Person Behind the Scientist
  Jonathan Kipnis and his lab members Noel Derecki and James Cronk authored the  recent Nature paper entitled Wild type microglia arrest pathology in a mouse model of Rett Syndrome. Watch the videos below for some insight into what makes them tick. Jonathan Kipnis, Ph.D. Noël Derecki, Ph.D. James Cronk
  
  
• Bone Marrow Transplants – Proceeding with Caution
  RETT SYNDROME RESEARCH TRUST WEBSITE The recent publication of the Kipnis paper in Nature has generated understandable excitement and questions in the Rett community. Email and Facebook are difficult vehicles for providing proper answers. Rett Syndrome is complex, and so is the research; this work doesn’t lend itself to sound bites. I know Rett mothers [...]
  
  
• Bone Marrow Transplant Stops the Development of Symptoms in Model of Rett Syndrome
  RETT SYNDROME RESEARCH TRUST WEBSITE Click Here to Read Press Release A paper published online today in the high-profile journal, Nature, describes the results of using a bone marrow transplant to dramatically stop the development of symptoms in pre-symptomatic male and female mouse models of Rett Syndrome.  The work was undertaken in the neuroimmunology laboratory [...]
  
  
• BioWorld Insight feature article on Rett Syndrome
  A recent issue of BioWorld Insight, the weekly newsletter that provides behind-the-scenes analysis and commentary on the biotechnology industry, included a feature article on Rett Syndrome.  The piece explored how recent interest in rare diseases on the part of pharmaceutical/biotech industry may impact research in disorders such as Rett Syndrome and Fragile X. Science, Rare Disease [...]
  
  
• Prof. Adrian Bird Gives Speech at London Event
  Professor Adrian Bird needs no introduction to anyone who follows Rett Syndrome research. His list of accomplishments includes discovering the MeCP2 protein, developing multiple animal models for the disease and authoring the seminal 2007 Science paper which introduced the startling concept that Rett Syndrome and other MECP2-related disorders are curable. He was the Guest of [...]
  
  
• Family’s Passion Spurs Research Success for Cystic Fibrosis
  RETT SYNDROME RESEARCH TRUST WEBSITE 02/10/2012 – Forbes.com Turning loss into hope, family offers inspiration — and a few lessons about drug discovery A poignant story in Thursday’s Boston Globe describes how the O’Donnell family of Boston channeled their love of a son, Joey, who died tragically at the age of 12 from cystic fibrosis, [...]
  
  
• What Success Looks Like
  Rett Syndrome Research Trust Website Doris Tulcin – A Mother’s Love Raises the Bar For All Non-Profits Half a century ago, a mother whose baby daughter was diagnosed with a life-threatening genetic disorder decided to fight it. Doris Tulcin is that mother, and Cystic Fibrosis is the disease against which she went to war. First [...]
  
  
• Letter from the Executive Director
  RETT SYNDROME RESEARCH TRUST WEBSITE Dear Friends, This October will mark fourteen years since my daughter, Chelsea, was officially diagnosed with Rett Syndrome. On that day I made my then two-year-old daughter a promise: I would do everything in my power to free her from Rett Syndrome. In pursuit of that promise I co-founded two [...]
  
  
• The X Factor
   RETT SYNDROME RESEARCH TRUST WEBSITE Those of you who follow the efforts of RSRT know that one of the treatment strategies we are pursuing is the reactivation of the MECP2 gene on the inactive X chromosome. A quick refresher for those in need of one: mutations in MECP2 cause Rett Syndrome (and a host of [...]