CUREUsher Research Strategy

Background
Usher syndrome is a complex rare genetic condition with many symptoms that primarily causes progressive sight loss leading to blindness, combined with varying level of deafness where in many cases it is also progressive. It is estimated that there are 400,000 people living with Usher syndrome worldwide with no cure or treatment to cure/halt any of the symptoms associated with Usher syndrome. There are three types of Usher syndrome 1, 2 and 3, each with different characteristics, and subtypes based on the mutated gene causing Usher syndrome. However, it is believed that many of the genetic mutations associated with Usher Syndrome have still yet to be identified due to Usher syndrome being poorly recognised or understood.

While hearing aids/cochlear implants are aids that may help provide some hearing function for those with Usher syndrome, they are not a complete solution as they do not restore fully hearing, and are therefore not considered a cure.

Our Vision and our Goals
At CUREUsher, our vision is simply a cure and/or treatment for all types of Usher syndrome. However, given the complex nature of Usher syndrome with many symptoms, our vision for a cure will be split into two areas, which focuses on the most severe symptoms of Usher syndrome:

  1. Our primary vision, is a cure and/or treatment for sight loss associated with Usher syndrome.
  2. Our secondary vision, is a cure and/or treatment for hearing loss associated with Usher syndrome.

To meet our primary vision, of curing and/or treating sight loss associated with Usher syndrome, the following are our goals:

  1.  To increase understanding and knowledge of sight loss associated with all types of Usher syndrome.
  2. To increase collaboration amongst scientific researchers internationally with the objective of identifying any knowledge gaps in the field of Usher syndrome, and of all its types.
  3. Provide funding to scientific research that aims to develop a cure or a treatment, which will either cure/halt the progression, for sight loss associated with Usher syndrome.
  4. Provide an accessible information portal to people affected by Usher syndrome, thus providing empowerment through knowledge.

To meet our secondary vision, of curing and/or treating hearing loss associated with Usher syndrome, the following are our goals:

  1. To increase knowledge and understanding of hearing loss associated with all types of Usher syndrome.
  2.  To increase collaboration amongst scientific researchers internationally with the objective of identifying any knowledge gaps in the field of Usher syndrome and of all its types.
  3. Provide funding to scientific research that aims to develop a cure or treatment, which will either cure/halt the progression, for hearing loss associated with Usher syndrome.
  4. Provide an accessible information portal to people affected by Usher syndrome thus providing empowerment through knowledge.

Delivering our goals:
CUREUsher aims to deliver its goals by, encouraging collaboration amongst scientific researchers internationally, and by providing financial grants/subsidies to medical research, which meets our ethical guidelines and research standards.

CUREUsher Research Strategy

a). Which Usher syndrome type
CUREUsher will provide financial grants/subsidies to research projects which target any of the Usher syndrome types, and research projects which target all types of Usher Syndrome. While the majority of scientific research mainly focuses on one symptom and type of Usher syndrome, due to the different mutated genes involved in each type of Usher syndrome, it should be noted that a successful outcome from a particular study on one type of Usher syndrome may potentially benefit further research on other types of Usher syndrome.

b) Allocation of research funding.
All high quality scientific research which aims to seek any of the following will be considered for funding:

  1. To increase knowledge and understanding of sight loss associated with Usher syndrome.
  2. A treatment that halts the progression of sight loss from any type of Usher syndrome.
  3. A cure that cures sight loss from any type of Usher syndrome.
  4. To increase knowledge and understanding of hearing loss associated with Usher syndrome.
  5. A treatment that halts the progression of hearing loss from any type of Usher syndrome.
  6. A cure that cures hearing loss from any type of Usher syndrome.

c) Types of Research
We aim to fund scientific research projects, from basic to clinical level, which are in line with our strategy and ethical standards.

d) Where can the research take place?
Due to the limited expertise and research on Usher syndrome in the UK, CUREUsher will provide funding to research in the UK and worldwide, that are eligible to be considered for funding if their aims are in line with our goals.

Ensuring Scientific Quality
The process for funding awarded varies differently. Some may adopt a two/three stage approach while some may include video submissions as part of the selection process. Any applicant who is eligible to be considered for funding will have their submission reviewed by our Scientific Advisory Panel. The Scientific Advisory Panel (SAP) will consist of scientific and medical experts with a deep knowledge and understanding of Usher syndrome and its symptoms. To ensure a fair and balanced view on all submissions, as condition of membership of the panel, all experts of the SAP will agree to a conflict of interest policy before accepting a position on the SAP.

Working in partnership
Collaborative partnerships will be considered with government agencies, charities, professional bodies and industry