Zoe Solsby

Research Grant Progress Report by Liang Feng, PhD, Stanford University

Researcher Liang Feng, Associate Professor of Molecular and Cellular Physiology at Stanford University, and Research Fellow Xue Guo received a grant award in 2020 to continue their work on the Molecular Mechanism of Cystinosis. The objectives of this study are to understand several key aspects of how membrane proteins that have important implications in cystinosis […]

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Stem Cell And Gene Therapy Webinar Update Video Now Available!

Stem Cell and Gene Therapy Trial Update with Dr. Stéphanie Cherqui, Jordan Janz, and Barb Kulyk Presented Tuesday, October 27, 2020 We had a record of 309 people participating from 20 countries around the world.  The participants represented the myriad of people in our community; adults with cystinosis, parents of children and adults with cystinosis,

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Final Report – Development and Characterization of a Rat Model of Cystinosis

In 2018, CRF awarded a grant to Olivier Devuyst, MD, PhD, University of Zurich, Switzerland, and Francesco Emma, MD, Bambino Gesù Pediatric Hospital, Rome, Italy, for their research on the Development and Characterization of a Rat Model of Cystinosis.  They successfully generated a novel Ctns-/- rat model that replicates the clinical symptoms of human nephropathic

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Research Progress Report by Jennifer Hollywood, PhD, and Alan Davidson, PhD, University of Auckland, New Zealand

The goal of this study is to conduct therapeutic drug intervention studies in Ctns knock-out rats to determine whether a combination treatment of cysteamine and Everolimus provides greater therapeutic benefit than cysteamine alone. CRF has granted research funding to the University of Auckland since 2009, awarding $805,143 for the important research conducted by Dr. Hollywood

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Research Progress Report by Michael Sekar, PhD, AMMA Therapeutics, Inc.

In the spring of 2020, CRF awarded a grant to AMMA Therapeutics for $304,064, the aim of their study is to develop a once-daily subcutaneous injection of cysteamine bitartate.  The project is led by Michael Sekar, PhD, Principal Investigator, and Laura Rita Rega, PhD, Co-Principal Investigator. The project is comprised of three specific Aims.  Aim

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Final Research Progress Report Ming Li, PhD and Jacob Kitzman, PhD, University of Michigan

The research study, “Dissect the Protein Turnover Mechanism of Cystinosis Mutants” received a CRF research grant of $285,648 in 2018.  Dr. Li explained in lay terms the purpose of the study,  “some people consider it as an “overkill” for grocery stores to throw away expired food even though most of them can still be consumed.

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