Research
Pipeline for Usher Syndrome Research (PUSH)
The Pipeline for Usher Syndrome Research (PUSH) at Boston Children’s Hospital brings together six world-class scientists dedicated to accelerating the development of treatments for Usher Syndrome (USH). PUSH aims to tackle multiple forms of Usher Syndrome in parallel, identifying subtypes and mutations that may be amenable to innovative therapies. By advancing potential therapies for many subtypes, PUSH will strive to identify optimized strategies that can move toward clinical trials, ultimately improving treatment options for hearing and vision loss associated with USH.
OUR WORLD CLASS SCIENTISTS LEADING PUSH RESEARCH
Usher syndrome is caused by mutations in multiple genes, each with many possible variants, so currently no single therapy will treat every patient. We created PUSH to develop treatments for multiple subtypes in parallel, including approaches that could help everyone with the same subtype, regardless of mutation. While there are several labs pursuing USH therapies around the world, PUSH is a unique opportunity because the six investigators are all leaders in their fields, are all located in one academic institution, and they all possess complementary expertise.
PUSH Updates:
The PUSH team achieved important scientific milestones across several USH targets
USH2A Therapeutic Progress:
Researchers achieved a key validation milestone using antisense technology to restore gene function in patient-derived cells, retinal and inner-ear organoids, and zebrafish models, while simultaneously advancing and optimizing additional antisense candidates to identify the strongest lead therapy for future clinical development.
USH3A Therapeutic Progress:
Researchers have chosen a target for USH3A, have collected samples, are developing IPSCs and have identified Gene replacement as a possible therapeutic approach. In vitro testing is in progress.
USH1B Therapeutic Progress:
Researchers have collected several USH1B samples, are developing IPSCs and have identified strategies for USH1B. In vitro testing is in progress.
USH2C Therapeutic Progress:
Researchers have collected samples, are developing IPSCs and have identified an ASO strategy for USH2C. In vitro testing is in progress.
Why Your Support Matters:
Your donation to the Usher Syndrome Society is crucial to continued support of this groundbreaking project at Boston Children’s Hospital. By supporting the Usher Syndrome Society, you will enable work that:
- Helps identify treatable forms of USH and advance targeted therapies.
- Supports the development of various therapeutic approaches, such as gene replacement, antisense oligonucleotides (ASOs), genome editing, small molecules, and others.
- Propels research toward clinical trials that could one day restore hearing and vision for those affected by USH.
Be Part of a Life-Changing Effort:
By contributing to the Usher Syndrome Society, you’ll help us support an initiative that accelerates the search for effective therapies. While not all forms of USH may have an immediate treatment, the project will significantly advance the knowledge and strategies needed to identify and treat those that can be helped.
Our commitment to this research at Boston Children’s Hospital will help transform lives by supporting research that has the potential to stop, and possibly even restore, hearing and vision loss. Every contribution to the Usher Syndrome Society will bring us closer to funding breakthrough treatments, offering hope and real change for the future of Usher syndrome.