Manton Center for Orphan Disease Research

The Manton Center is proud to announce our recent awardees! Congratulations to all!

2018 Manton Center Fellowships:

Mehdi Pirouz, PhD
Mentor: Richard Gregory
Project title: Role of Dis3l2 Exoribonuclease in the Pediatric Perlman Syndrome

Sara Bizzotto, PhD
Mentor: Christopher Walsh, MD, PhD
Project title: Exploring new genetic causes and pathological mechanisms of epileptic focal cortical dysplasia

Zhongjie Fu, PhD
Mentor: Lois Smith, MD, PhD
Project title: FGF21 protects against retinal degeneration in retinitis pigmentosa

Neha Nagpal, PhD
Mentor: Suneet Agarwal, MD, PhD
Project title: Mechanisms and therapeutic targeting of non-coding RNA dysregulation in telomere diseases

2018 Pilot Project Awards:

Boxun Zhao, PhD
Mentor: Alice Lee, PhD and Tim Yu, MD, PhD
Project title: Pathogenic structural variant identification and splicing defect correction

Lucia Ambrosio, MD, PhD
Mentor: Anne Fulton, MD
Project title: Translational Read-Through Inducing Drugs (TRIDS) to treat Inherited Retinal Disorders (IRDs)

The Manton Center Goals

The Manton Center will be the home for physicians and scientists who share a common vision. The goals of this collaboration will be to:

  • Unlock the mysteries of orphan diseases in order to develop better diagnostic tests and design new treatments and cures
  • Discover fundamental biological principles that have broad applications and can lead to advances in our understanding of common diseases such as cancer, heart disease, diabetes and other major health problems
  • Train a new generation of investigators who will infuse creative energy into the battle against orphan diseases
  • Disseminate The Manton Center’s findings among lay and medical/ scientific communities locally, nationally and globally to spur interest and support for orphan disease research and care