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Abuddayeh-Gootenberg Lab

Adrian Bird, PhD

Angelman syndrome

Apertura Gene Therapy

Baylor College of Medicine

Beam Therapeutics

Blood Brain Barrier

Bone Marrow Transplant

Boston Children’s Hospital

Boys and Rett Syndrome

Brian Kaspar, PhD

Broad Institute

Business Development

CURE 360 Webinar

Case Western Reserve University

Children’s Hospital at Montefiore

Clinical Initiatives

Clinical Trials

Cystic Fibrosis

Deep Brain Stimulation

Digital Natural History Study

Duchenne muscular dystrophy

Erik Sontheimer. PhD

Gail Mandel, PhD

Gastrointestinal

Gene Replacement

Gene Therapy Consortium

Genetic Engineers

Guoping Feng, PhD

Harvard Medical School

Huda Zoghbi, MD

Jeannie Lee, MD, PhD

Jennifer Doudna, PhD

Jonathan Gootenberg, PhD

Jonathan Watts, PhD

MECP2 Consortium

MECP2 Duplication Fund

MECP2 Editing Consortium

MECP2 duplication syndrome

MECP2 reactivation

Michael Greenberg, PhD

Milken Institute

Missense Mutations

Monica Justice, PhD

Nationwide Children’s Hospital

Non-Viral Delivery

Ohio State University

Omar Abuddayeh, PhD

Outcome Measures

Protein Replacement

RNA Trans-splicing

RSRT Ambassadors

Randy Carpenter, MD

Related Disorders

Rett Global Registry

Rett Syndrome Clinic Atlanta

Rett Syndrome Global Registry

Reverse Rett LA

Reverse Rett NYC

Roadmap to Cures

Roadmap to Cures 2.0

Roadmap to a Cure 1.0

Scot Wolfe, PhD

Shape Therapeutics

Simons Foundation

Steve Gray, PhD

Stuart Cobb, PhD

The Jackson Laboratory

Translational Medicine Advisory Board

Tri-State Rett Syndrome Center

Understanding MECP2

University of Edinburgh

University of North Carolina at Chapel Hill

University of Siena

VIBRANT Program

Vico Therapeutics

Wave Life Sciences

scientific initiatives

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Roadmap to Cures

$40M