Exploring the Link Between MeCP2 and Gut Physiology to Test a Novel Probiotic Therapy for Rett Syndrome
Ali Khoshnan, PhD & Sarkis K. Mazmanian, PhD | California Institute of Technology
$200,000 AWARDED
This project is testing the concept that neurodevelopmental and behavioral abnormalities in Rett may be linked to GI defects and/or disruption in the homeostasis of gut microbiota.
Current Projects
-
Rett Syndrome Microbiome Study for the Advancement… Beth McCormick
$481,774READ MORE -
Passive Monitoring of Rett Patients with the… Emerald Pilot and MC10 nPoint Development Study
$164,670READ MORE -
Rett Syndrome Biorepository Coriell Institute for Medical Research
$135,000READ MORE -
The generation and phenotypic assessment of mouse… Jackson Labs
$417,690READ MORE -
RNA editing for MECP2 mutations via RESTORE Thorsten Stafforst, PhD
$366,520READ MORE -
Correction of MECP2 Mutations with Engineered… Joseph Jacobson, PhD
$50,000READ MORE -
Development of a cDNA/mRNA Delivery System…
$68,000READ MORE -
Human In Vitro Models For X Chromosome… Joost Gribnau, PhD
$401,000READ MORE -
Development of siRNA-based compounds to potently… Anastasia Khvorova, PhD
$435,515READ MORE -
Gene Editing Approach to Remove Duplicated Region Ronald Cohn, MD | The Hospital for Sick Children
$570,000READ MORE -
A Drug-Screening Platform for the MECP2… Alysson Renato Muotri, PhD | University of California San Diego
$792,000READ MORE -
A Study to Evaluate Ketamine for the Treatment of… Clinical Trial Consortium
$1,300,000READ MORE -
Triheptanoin (UX007) | Treatment of Mitochondrial… Daniel Tarquinio, DO, MS | Center for Rare Neurological Diseases
$51,600READ MORE -
Protein Replacement for Rett Syndrome Armagen
$125,000READ MORE -
Gene Therapy Approach to Treating MECP2… Kevin Foust, PhD | Ohio State University
$40,000READ MORE -
Optimizing Gene Therapy for Rett Syndrome Kathrin Meyer, PhD
$221,004READ MORE -
A System For Dosage-independent Control of Mecp2… Michael Elowitz, PhD | Caltech
$197,681READ MORE -
Developing a pre-clinical DNA base editing program… BEAM Therapeutics
$1,870,660READ MORE -
Exploration of the Impact of Cyclodextrin on… Stephen Turley, PhD & Adam Lopez, PhD | University of Texas Southwestern…
$156,180READ MORE -
Investigating the Potential of Antisense… Huda Zoghbi, MD | Baylor College of Medicine
$530,000READ MORE -
Identification of Gene Modifiers that Ameliorate… Monica Justice, PhD | Hospital for Sick Children (Toronto)
$2,300,000READ MORE -
Identifying Therapeutics for Treating Rett… Michael Greenberg | Harvard University
$110,000READ MORE -
Development of an In-Vitro Cell System For… Q-State BioSciences
$828,141READ MORE -
Modeling MECP2 Dosage in Human Cerebral Organoids Alysson Renato Muotri, PhD | University of California San Diego
$209,000READ MORE -
RNA and Genome editing for treatment of Rett… Jonathan Watts, PhD / Erik Sontheimer, PhD / Scot Wolfe, PhD / Anastasia…
$2,403,735READ MORE -
Discovery of Compounds Promoting MECP2… Andrew Napper, PhD | Nemours/A.I. duPont Hospital for Children
$268,452READ MORE -
Reversal of Rett Phenotype: A screen for compounds… Rudolf Jaenisch, MD | Whitehead Institute
$180,000READ MORE -
A Forward Genetic Screen to Identify Druggable… Huda Zoghbi, MD | Baylor College of Medicine
$1,485,949READ MORE -
Identification of Genetic Modifiers in Rett… Jeffrey Neul, MD, PhD | University of California San Diego
$315,000READ MORE -
RNA-editing as a gene therapy approach for Rett… Guoping Feng, PhD /Feng Zhang, PhD /Robert Desimone, PhD MIT/Broad/Harvard
$2,332,000READ MORE -
New Editing Enzymes for RNA Gail Mandel, PhD | John Sinnamon, PhD
$345,000READ MORE -
From Sensory-Perceptual Representations to… John Foxe, PhD., University of Rochester, Albert Einstein College of…
$533,607READ MORE -
Treatment of Rett Syndrome with Lovastatin Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine,…
$403,000READ MORE -
Is MECP2 Duplication/Triplication Syndrome… Huda Zoghbi, MD | Baylor College of Medicine
$236,000READ MORE -
Screening for drugs that can rebalance long gene… Mark Zylka, PhD | University of North Carolina at Chapel Hill
$400,000READ MORE -
New Molecular Tools for Directed Editing of MeCP2… Peter Beal, PhD | UC Davis
$563,870READ MORE -
Outlining the Autonomic Signature of Rett Syndrome Debra Weese-Mayer, MD & Michael Carroll, PhD | Ann & Robert H. Lurie…
$157,000READ MORE -
Therapeutic Approaches to Reversing Forebrain and… David Katz, PhD | Case Western Reserve University
$500,000READ MORE -
Treatment of Rett Syndrome with Copaxone Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine,…
$487,000READ MORE -
PNA Nanoparticles for Gene Editing of Rett… Peter Glazer, MD, PhD & Mark Saltzman, PhD | Yale University
$275,000READ MORE -
Clinical Development of NLX-101 in Rett Syndrome;… Mark Varney, PhD | Neurolixis
$585,000READ MORE -
Systems Genetics Approach toward Understanding… Terry Magnuson, PhD | University of North Carolina at Chapel Hill
$200,000READ MORE -
RNA trans-splicing therapy in Rett Syndrome Stuart Cobb, PhD and Chris Sibley, PhD | University of Edinburgh
$1,158,475READ MORE -
High-Content Phenotypic Screening of Existing… Christopher Gibson, PhD & Dean Li, MD, PhD | Recursion Pharmaceuticals
$50,000READ MORE -
Readthrough of premature termination codons for… Allan Jacobson, PhD / Jonathan Watts, PhD | UMASS
$323,000READ MORE -
Exploring the Link Between MeCP2 and Gut… Ali Khoshnan, PhD & Sarkis K. Mazmanian, PhD | California Institute of…
$200,000READ MORE -
Testing NR2A and NR2B NAMs in the mouse models of… Michela Fagiolini, PhD | Boston Children’s Hospital
$464,336READ MORE -
Reactivation of MECP2 and CDKL5 genes by… Andrea Cerase, PhD | Queen Mary University of London
$351,022READ MORE -
Testing Whether LM22A-4 Improves Hippocampal… Lucas Pozzo-Miller, PhD | University of Alabama Birmingham
$110,000READ MORE -
Reactivation of MECP2 by Artificial Transcription… Raf Schmid, PhD, MSCR | University of Pennsylvania
$141,912READ MORE -
Preclinical Studies of LM22A-4 in Mouse Models of… David Katz, PhD | Case Western Reserve University
$272,000READ MORE -
Reactivation of MECP2 with Epigenome Editing Tools… Rudolf Jaenisch, MD | Whitehead Institute
$600,000READ MORE -
Evaluating Cell-permeant Miniature Proteins… Alanna Schepartz, PhD | Yale University
$270,216READ MORE -
Outcome Measures and Biomarkers Development Timothy Benke, MD, PhD | Aleksandra Djukic, MD, PhD | Alan Percy, MD |…
$4,500,000READ MORE -
Gene Therapy Consortium 1.0 Gail Mandel, PhD | Stuart Cobb, PhD | Steven Grey, PhD
$1,600,000READ MORE -
Gene Therapy Consortium 2.0 Adrian Bird, PhD | Stuart Cobb, PhD | James Wilson, MD, PhD
$5,463,184READ MORE -
Outcome Measures and Biomarkers Development Timothy Benke, MD, PhD | Alan Percy, MD | Daniel Tarquinio, DO, MS
$4,500,000READ MORE -
MECP2 Consortium Adrian Bird, PhD | Michael Greenberg, PhD | Gail Mandel, PhD
$9,000,000READ MORE -
Clinical Trial Consortium David Lieberman & Mustafa Sahin | Eric Marsh | Jeff Neul | Alan Percy |…
$2,442,507READ MORE -
Reactivating MECP2 Consortium Ben Philpot, PhD & Bryan Roth, PhD | Antonio Bedalov, MD, PhD | Jeannie…
$6,416,072READ MORE