Funded Research Through RS Angels
Every step forward in Rett syndrome research begins with families who refuse to give up hope. Through community support and relentless commitment, RS Angels has helped fund critical studies aimed at understanding the condition and advancing real treatment possibilities. This work reflects a shared belief that progress is possible, and that brighter futures are within reach for every child affected.
Note: a couple of organizational acronyms to recognize:
- RSO = rettsyndrome.org (2021, changed name to IRSF)
- RSRT = Rett Syndrome Research Trust
YEAR
Funded Research
CONTRIBUTION
2000-2010
Donations to several research projects (IRSF / RSRT)
$150,000
2011
IGF-1 Clinical Trial Boston Rett Program (BCH)
$20,000
2012
IGF-1 Clinical Trial Boston Rett Program
$115,000
2013
1. Yun Li, The Whitehead Institute, MIT – “Modeling Rett syndrome using TALEN technology in human pluripotent stem cells” (RSRT)
2. IGF-1 Clinical Trial Boston Rett Program (IRSF)
$100,000
$25,000
2014
1. Dr. Michela Fagiolini, BCH: “Assessing NMDAr Modulators to Ameliorate Cortical Regression in Rett Syndrome” (IRSF)
2. Dr. Jeannie Lee, Harvard Medical: An Oligotherapeutics (RSRT) Approach to Treat Rett Syndrome (RSRT)
3. Rettland Foundation, (direct to Foundation)
$150,000
$20,000
$12,000
2015
IGF-1 Clinical Trial at Boston Children’s Hospital: (IRSF)
$85,000
2016
1. Dr. Michaela Fagiolini, Boston Children’s Hospital: “Testing NR2A and NR2B Modulators in Rett Mouse Models” (RSRT)
2. Dr. Timothy Hammond, Boston Children’s Hospital: “Microglia contribute to circuit defects in Mecp2 null mice independent of microglia-specific loss of Mecp2 expression” (IRSF)
$41,000
$41,000
2017
1. Jeannie Lee, MD, PhD, Massachusetts General Hospital, Harvard: “Reactivating the silent MECP2 allele through a synergistic drug mechanism” (IRSF / RSRT)
2. FOXG1 project at Boston Children’s Hospital (BCH)
3. Rett Clinic at Boston Children’s Hospital for MyTobi stand (BCH)
$74,000
$82,000
$2,000
2018
1. Dr. Charles Nelson, Boston Children’s Hospital: Phase 3 Clinical Trial, trofinetide (IRSF)
2. Dr. Rudolph Jaenisch Lab at Whitehead Institute, Cambridge, Reactivation of MECP2 Using CRISPR (RSRT)
$35,000
$35,000
2019
1. Dr. Jonathan Watts, University of Mass Medical: “MECP2 mutations, DNA and RNA editing approaches” (RSRT)
2. Clinical Network through IRSF to benefit the Boston Children’s Rett Program (IRSF)
$25,000
$25,000
2020
1. Guoping Feng, PhD, MIT: “RNA Editing” (RSRT)
2. Boston Children’s Hospital Rett Syndrome Program: “Natural History Study Database Support and Ongoing Data Collection” (RSO)
$15,000
$15,000
2021
1. Dr. Michaela Fagiolini: Boston Children’s Hospital, “Targeting choroid plexus Otx2 as novel therapy in RTT” (IRSF)
2. Rett Clinic at Boston Children¹s Hospital, “Ketamine Study / Global Registry support” (RSRT)
$20,000
$20,000
2022
1. Dr. Michaela Fagiolini: Boston Children’s Hospital, “Targeting choroid plexus Otx2 as novel therapy in RTT” (IRSF)
2. Rett Clinic at Boston Children¹s Hospital (RSRT)
$20,000
$20,000
2023
Dr. Michaela Fagiolini: Rett research given directly through the Boston Children’s Hospital Trust: “Visual impairment associated with RTT as a tool to study the disease” (directly to BCH)
$25,000
2024
1. Omar Abudayyeh and Jonathan Gootenberg, MIT / Harvard: “RNA transsplicing” (RSRT)
2.Jonathan Watts, Erik Sontheimer and Scot Wolfe, University of Massachusetts Medical School: “Base and Prime Editing Approaches for Rett Syndrome” (RSRT)
$25,000
$25,000
2025
QurCan / UMASS: “Developing Non-Viral Delivery for Rett Syndrome Gene Therapy” (RSRT)