A Forward Genetic Screen to Identify Druggable Modulators of MECP2 Levels

Current Projects

• CURE
• TREAT
• ENABLE
• LEARN
• Past Projects
• Consortia
• DNA/RNA/Protein Therapy
• MECP2 Duplication

• 
  Reactivation of MECP2 with Epigenome Editing Tools… Rudolf Jaenisch, MD | Whitehead Institute
  $600,000
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• 
  Identifying Therapeutics for Treating Rett… Michael Greenberg | Harvard University
  $110,000
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• 
  Development of an In-Vitro Cell System For… Q-State BioSciences
  $330,000
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• 
  Reversal of Rett Phenotype: A screen for compounds… Rudolf Jaenisch, MD | Whitehead Institute
  $180,000
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• 
  Protein Replacement for Rett Syndrome Armagen
  $125,000
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• 
  Spliceosome-Mediated RNA Trans-Splicing Therapy… Stuart Cobb, PhD | University of Glasgow
  $627,012
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• 
  Modeling MECP2 Dosage in Human Cerebral Organoids Alysson Renato Muotri, PhD | University of California San Diego
  $209,000
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• 
  A Drug-Screening Platform for the MECP2… Alysson Renato Muotri, PhD | University of California San Diego
  $792,000
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• 
  Triheptanoin (UX007) | Treatment of Mitochondrial… Daniel Tarquinio, DO, MS | Emory University | Children's Pediatric…
  $51,600
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• 
  Outcome Measures and Biomarkers Development Timothy Benke, MD, PhD | Aleksandra Djukic, MD, PhD | Alan Percy, MD |…
  $4,500,000
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• 
  From Sensory-Perceptual Representations to… John Foxe, PhD., University of Rochester, Albert Einstein College of…
  $533,607
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• 
  Exploration of the Impact of Cyclodextrin on… Stephen Turley, PhD & Adam Lopez, PhD | University of Texas Southwestern…
  $156,180
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• 
  Discovery of Compounds Promoting MECP2… Andrew Napper, PhD | Nemours/A.I. duPont Hospital for Children
  $268,452
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• 
  Treatment of Rett Syndrome with Lovastatin Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine,…
  $403,000
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• 
  Gene Therapy Approach to Treating MECP2… Kevin Foust, PhD | Ohio State University
  $40,000
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• 
  Investigating the Potential of Antisense… Huda Zoghbi, MD | Baylor College of Medicine
  $530,000
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• 
  A Forward Genetic Screen to Identify Druggable… Huda Zoghbi, MD | Baylor College of Medicine
  $733,000
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• 
  Is MECP2 Duplication/Triplication Syndrome… Huda Zoghbi, MD | Baylor College of Medicine
  $236,000
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• 
  Outlining the Autonomic Signature of Rett Syndrome Debra Weese-Mayer, MD & Michael Carroll, PhD | Ann & Robert H. Lurie…
  $157,000
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• 
  Exploring the Link Between MeCP2 and Gut… Ali Khoshnan, PhD & Sarkis K. Mazmanian, PhD | California Institute of…
  $200,000
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• 
  Testing NR2A and NR2B NAMs in the mouse models of… Michela Fagiolini, PhD | Boston Children’s Hospital
  $464,336
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• 
  Therapeutic Approaches to Reversing Forebrain and… David Katz, PhD | Case Western Reserve University
  $500,000
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• 
  Testing Whether LM22A-4 Improves Hippocampal… Lucas Pozzo-Miller, PhD | University of Alabama Birmingham
  $110,000
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• 
  Preclinical Studies of LM22A-4 in Mouse Models of… David Katz, PhD | Case Western Reserve University
  $272,000
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• 
  Screening for drugs that can rebalance long gene… Mark Zylka, PhD | University of North Carolina at Chapel Hill
  $400,000
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• 
  Clinical Development of NLX-101 in Rett Syndrome;… Mark Varney, PhD | Neurolixis
  $585,000
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• 
  Systems Genetics Approach toward Understanding… Terry Magnuson, PhD | University of North Carolina at Chapel Hill
  $200,000
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• 
  Identification of Genetic Modifiers in Rett… Jeffrey Neul, MD, PhD | University of California San Diego
  $315,000
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• 
  Identification of Gene Modifiers that Ameliorate… Monica Justice, PhD | Hospital for Sick Children (Toronto)
  $2,300,000
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• 
  Treatment of Rett Syndrome with Copaxone Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine,…
  $487,000
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• 
  Low-dose Ketamine for the Treatment of Rett… David M. Katz, PhD, Case Western Reserve University & Dan Sessler, MD,…
  $1,295,131
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• 
  Reactivating MECP2 Consortium Ben Philpot, PhD & Bryan Roth, PhD | Antonio Bedalov, MD, PhD | Jeannie…
  $6,416,072
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• 
  MECP2 Consortium Adrian Bird, PhD | Michael Greenberg, PhD | Gail Mandel, PhD
  $9,000,000
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• 
  Gene Therapy Consortium Stuart Cobb, PhD | Steven Gray, PhD | Gail Mandel, PhD | Alysson Muotri,…
  $3,595,265
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• 
  Outcome Measures and Biomarkers Development Timothy Benke, MD, PhD | Aleksandra Djukic, MD, PhD | Alan Percy, MD |…
  $4,500,000
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