Exploring the Link Between MeCP2 and Gut Physiology to Test a Novel Probiotic Therapy for Rett Syndrome

Current Projects

• CURE
• TREAT
• ENABLE
• LEARN
• Past Projects
• Clinical Trials & Studies
• Consortia
• DNA/RNA/Protein Therapy
• MECP2 Duplication

• 
  Ketamine Clinical Trial Jeffrey Neul, MD, PhD
  $636,000
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• 
  Rett Syndrome Microbiome Study for the Advancement… Beth McCormick
  $481,774
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  Passive Monitoring of Rett Patients with the… Emerald Pilot and MC10 nPoint Development Study
  $164,670
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• 
  Rett Syndrome Biorepository Coriell Institute for Medical Research
  $135,000
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• 
  The generation and phenotypic assessment of mouse… Jackson Labs
  $417,690
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  RNA editing for MECP2 mutations via RESTORE Thorsten Stafforst, PhD
  $366,520
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• 
  Correction of MECP2 Mutations with Engineered… Joseph Jacobson, PhD
  $50,000
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• 
  Development of a cDNA/mRNA Delivery System…
  $68,000
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  Human In Vitro Models For X Chromosome… Joost Gribnau, PhD
  $401,000
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  Development of siRNA-based compounds to potently… Anastasia Khvorova, PhD
  $435,515
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  Interrogation of Genome Editing Strategies as a… Ronald Cohn, MD | The Hospital for Sick Children
  $570,000
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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 | Center for Rare Neurological Diseases
  $51,600
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• 
  Protein Replacement for Rett Syndrome Armagen
  $125,000
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• 
  Gene Therapy Approach to Treating MECP2… Kevin Foust, PhD | Ohio State University
  $40,000
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• 
  Optimizing Gene Therapy for Rett Syndrome Kathrin Meyer, PhD
  $221,004
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• 
  A System For Dosage-independent Control of Mecp2… Michael Elowitz, PhD | Caltech
  $197,681
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• 
  Developing a pre-clinical DNA base editing program… BEAM Therapeutics
  $1,870,660
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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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• 
  Investigating the Potential of Antisense… Huda Zoghbi, MD | Baylor College of Medicine
  $530,000
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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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• 
  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
  $828,141
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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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• 
  RNA and Genome editing for treatment of Rett… Jonathan Watts, PhD / Erik Sontheimer, PhD / Scot Wolfe, PhD / Anastasia…
  $2,403,735
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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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• 
  Reversal of Rett Phenotype: A screen for compounds… Rudolf Jaenisch, MD | Whitehead Institute
  $180,000
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• 
  A Forward Genetic Screen to Identify Druggable… Huda Zoghbi, MD | Baylor College of Medicine
  $1,485,949
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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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• 
  RNA-editing as a gene therapy approach for Rett… Guoping Feng, PhD /Feng Zhang, PhD /Robert Desimone, PhD MIT/Broad/Harvard
  $2,332,000
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• 
  New Editing Enzymes for RNA Gail Mandel, PhD | John Sinnamon, PhD
  $345,000
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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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  Treatment of Rett Syndrome with Lovastatin Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine,…
  $403,000
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• 
  Is MECP2 Duplication/Triplication Syndrome… Huda Zoghbi, MD | Baylor College of Medicine
  $236,000
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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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• 
  New Molecular Tools for Directed Editing of MeCP2… Peter Beal, PhD | UC Davis
  $563,870
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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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  Therapeutic Approaches to Reversing Forebrain and… David Katz, PhD | Case Western Reserve University
  $500,000
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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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  PNA Nanoparticles for Gene Editing of Rett… Peter Glazer, MD, PhD & Mark Saltzman, PhD | Yale University
  $275,000
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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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  RNA trans-splicing therapy in Rett Syndrome Stuart Cobb, PhD and Chris Sibley, PhD | University of Edinburgh
  $1,158,475
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  High-Content Phenotypic Screening of Existing… Christopher Gibson, PhD & Dean Li, MD, PhD | Recursion Pharmaceuticals
  $50,000
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  Readthrough of premature termination codons for… Allan Jacobson, PhD / Jonathan Watts, PhD | UMASS
  $323,000
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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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  Reactivation of MECP2 and CDKL5 genes by… Andrea Cerase, PhD | Queen Mary University of London
  $351,022
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  Testing Whether LM22A-4 Improves Hippocampal… Lucas Pozzo-Miller, PhD | University of Alabama Birmingham
  $110,000
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  Reactivation of MECP2 by Artificial Transcription… Raf Schmid, PhD, MSCR | University of Pennsylvania
  $141,912
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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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  Reactivation of MECP2 with Epigenome Editing Tools… Rudolf Jaenisch, MD | Whitehead Institute
  $600,000
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  Evaluating Cell-permeant Miniature Proteins… Alanna Schepartz, PhD | Yale University
  $270,216
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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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  Gene Therapy Consortium 1.0 Gail Mandel, PhD | Stuart Cobb, PhD | Steven Grey, PhD
  $1,600,000
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  Gene Therapy Consortium 2.0 Adrian Bird, PhD | Stuart Cobb, PhD | James Wilson, MD, PhD
  $5,463,184
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  Outcome Measures and Biomarkers Development Timothy Benke, MD, PhD | Alan Percy, MD | Daniel Tarquinio, DO, MS
  $4,500,000
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  MECP2 Consortium Adrian Bird, PhD | Michael Greenberg, PhD | Gail Mandel, PhD
  $12,200,000
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  Clinical Trial Consortium David Lieberman & Mustafa Sahin | Eric Marsh | Jeff Neul | Alan Percy |…
  $2,442,507
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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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