A Forward Genetic Screen to Identify Druggable Modulators of MECP2 Levels
Huda Zoghbi, MD | Baylor College of Medicine
$733,000 AWARDED
Dr. Zoghbi will screen compounds in search of any that can reduce levels of MeCP2 for the duplication/triplication syndrome. Any positive “hits” could form the foundation for drug discovery efforts.
Current Projects
-
Reactivation of MECP2 and CDKL5 genes by… Andrea Cerase, PhD | Queen Mary University of London
$351,022READ MORE -
Optimizing Gene Therapy for Rett Syndrome Kathrin Meyer, PhD
$221,004READ MORE -
Reactivation of MECP2 by Artificial Transcription… Ben Philpot, PhD | University of North Carolina
$141,912READ MORE -
Reactivation of MECP2 with Epigenome Editing Tools… Rudolf Jaenisch, MD | Whitehead Institute
$600,000READ 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 -
Reversal of Rett Phenotype: A screen for compounds… Rudolf Jaenisch, MD | Whitehead Institute
$180,000READ MORE -
Protein Replacement for Rett Syndrome Armagen
$125,000READ MORE -
Spliceosome-Mediated RNA Trans-Splicing Therapy… Stuart Cobb, PhD | University of Edinburgh
$918,475READ MORE -
Modeling MECP2 Dosage in Human Cerebral Organoids Alysson Renato Muotri, PhD | University of California San Diego
$209,000READ MORE -
A Drug-Screening Platform for the MECP2… Alysson Renato Muotri, PhD | University of California San Diego
$792,000READ MORE -
Triheptanoin (UX007) | Treatment of Mitochondrial… Daniel Tarquinio, DO, MS | Center for Rare Neurological Diseases
$51,600READ MORE -
Outcome Measures and Biomarkers Development Timothy Benke, MD, PhD | Aleksandra Djukic, MD, PhD | Alan Percy, MD |…
$4,500,000READ MORE -
From Sensory-Perceptual Representations to… John Foxe, PhD., University of Rochester, Albert Einstein College of…
$533,607READ MORE -
Exploration of the Impact of Cyclodextrin on… Stephen Turley, PhD & Adam Lopez, PhD | University of Texas Southwestern…
$156,180READ MORE -
Discovery of Compounds Promoting MECP2… Andrew Napper, PhD | Nemours/A.I. duPont Hospital for Children
$268,452READ MORE -
Treatment of Rett Syndrome with Lovastatin Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine,…
$403,000READ MORE -
Gene Therapy Approach to Treating MECP2… Kevin Foust, PhD | Ohio State University
$40,000READ MORE -
Investigating the Potential of Antisense… Huda Zoghbi, MD | Baylor College of Medicine
$530,000READ MORE -
A Forward Genetic Screen to Identify Druggable… Huda Zoghbi, MD | Baylor College of Medicine
$733,000READ MORE -
Is MECP2 Duplication/Triplication Syndrome… Huda Zoghbi, MD | Baylor College of Medicine
$236,000READ MORE -
Outlining the Autonomic Signature of Rett Syndrome Debra Weese-Mayer, MD & Michael Carroll, PhD | Ann & Robert H. Lurie…
$157,000READ 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 -
Therapeutic Approaches to Reversing Forebrain and… David Katz, PhD | Case Western Reserve University
$500,000READ MORE -
Testing Whether LM22A-4 Improves Hippocampal… Lucas Pozzo-Miller, PhD | University of Alabama Birmingham
$110,000READ MORE -
Preclinical Studies of LM22A-4 in Mouse Models of… David Katz, PhD | Case Western Reserve University
$272,000READ MORE -
Screening for drugs that can rebalance long gene… Mark Zylka, PhD | University of North Carolina at Chapel Hill
$400,000READ 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 -
Identification of Genetic Modifiers in Rett… Jeffrey Neul, MD, PhD | University of California San Diego
$315,000READ MORE -
Identification of Gene Modifiers that Ameliorate… Monica Justice, PhD | Hospital for Sick Children (Toronto)
$2,300,000READ MORE -
Treatment of Rett Syndrome with Copaxone Aleksandra Djukic, MD, PhD Albert Einstein College of Medicine,…
$487,000READ MORE -
Low-dose Ketamine for the Treatment of Rett… David M. Katz, PhD, Case Western Reserve University & Dan Sessler, MD,…
$1,295,131READ MORE -
Clinical Trial Consortium David Lieberman & Mustafa Sahin | Eric Marsh | Jeff Neul | Alan Percy |…
$2,367,715READ MORE -
Reactivating MECP2 Consortium Ben Philpot, PhD & Bryan Roth, PhD | Antonio Bedalov, MD, PhD | Jeannie…
$6,416,072READ MORE -
MECP2 Consortium Adrian Bird, PhD | Michael Greenberg, PhD | Gail Mandel, PhD
$9,000,000READ MORE -
Gene Therapy Consortium Stuart Cobb, PhD | Steven Gray, PhD | Gail Mandel, PhD | Alysson Muotri,…
$4,707,696READ MORE -
Outcome Measures and Biomarkers Development Timothy Benke, MD, PhD | Alan Percy, MD | Daniel Tarquinio, DO, MS
$4,500,000READ MORE
