The Urman Family - Striving for Independence

Sam and Shani Urman reside in Toronto, Canada, with their five children. It’s hard not to smile when you speak with Shani Urman. She is a bright and cheerful woman who speaks joyfully about her children, no matter what challenges they may face. Four of the Urman children live with Usher 1F. 

Shani serves up realistic advice with a dash of optimism. “You don’t have to be as good as everybody, be as good as you need to be for you,” she says. This is a healthy reminder for anyone to internalize. And what was it that her family, her children, needed so they could “be as good as they needed to be” with these different constraints of COVID? The quick shift meant that there would be no more in-person classes. Shani found that instead of video, her children would resume classes over the phone. The transition was relatively seamless, as her children have received cochlear implants.

One thing that every parent worries about is the safety and security of their children. The Urmans are no different. There is a general fear of safety that is heightened for parents of a child living with Usher 1F.

Read more of the Urmans' story

For those of you who might be considering your end-of-the-year gift planning, Usher 1F Collaborative is set up to accept gifts of stock. Please contact us for details. We are also appreciative of gifts from IRAs during and/or after the CARES Act.

Announcing Our Ten to Cure Initiative

The Usher 1F community is excited to announce a new major gift initiative called “Ten to Cure!” The name alludes to the campaign goal of raising $10 Million in new money to catapult research to the next critical levels for new breakthroughs.
           
Through several weeks of research, development, planning, and execution, the “Ten to Cure” initiative is ready for launch! Prospective donors have been identified, and strategic plans for the trajectory of each have been created. The team will be working with members of our board to network with donors in your geographic area and develop those relationships. Through this multi-city reach-out model, we aim to utilize various methods to present our case to potential donors. Considering the impact of the Coronavirus, we plan to supplement local networking with virtual gatherings. We look forward to hosting you for virtual information sessions, and we look forward to you sharing these virtual information sessions with your network as well.
           
We owe a tremendous debt of gratitude to Berman Branding, founded and run by Rebecca Berman (friend of Jared & Rachel Root), who crafted a profoundly compelling visual brochure for us. Rebecca is also the same person who created our new Usher 1F Collaborative logo! Our brochure is a digital document which can also be printed and delivered as a stunning presentation. 

Read more about Ten to Cure

Our University of Oregon Team Perseveres Through Two Incredible Challenges

Satellite map showing proximity of fire (red) to our Usher 1F lab at the University of Oregon (white arrow)

The workplace shutdowns brought about between March and May by the COVID pandemic also included research laboratories at the universities where our Usher 1F research labs are housed. While COVID-19 has impacted all of our research labs, one of our labs, the University of Oregon Institute of Neuroscience in Eugene, Oregon, was affected by yet a second disaster, the out-of-control wildfires on the west coast.
 
Read about their experiences coping with both disasters

Research Updates

David Corey, PhD, Harvard Medical School, presented his Usher 1F work at the Usher Syndrome Coalition virtual conference in July 2020. He described his work developing a mini gene, that is a gene with pieces not essential for vision removed so that the entire remaining gene would fit on the conventional delivery method for gene therapy, an adeno-associated virus (AAV). We are excited that this approach is restoring hearing in a newborn Usher 1F mouse, and Dr. Corey is now testing the mini gene on our Usher 1F zebrafish that he obtained from our University of Oregon team to see if it restores vision. Please watch his presentation.
 
Zubair Ahmed, PhD, University of Maryland, is in talks with two biopharma companies, the first to determine if their compound, a translational read through inducing drug, will be effective in restoring vision in those with Usher 1F. He is in talks with a second biopharma company about testing their state-of-the-art approach to gene therapy using dual AAVs with inteins.
 
Monte Westerfield, PhD, and Jennifer Phillips, PhD, University of Oregon Institute of Neuroscience, are collaborating with a Dutch Usher Syndrome research team, headed by Erwin van Wyk, PhD, Raboud University Medical Center, to test exon skipping for Usher 1F using antisense oligonucleotides. This research is similar to a treatment from ProQR already in clinical trial for Usher Syndrome type 2A. 

Chan Zuckerberg Initiative Rare As One Project Grant

Rare As One grantees (Usher 1F top left)

On November 25, 2019, we received an email with the exciting words,
 
“We are very pleased to inform you that your application will be recommended for funding from the Chan Zuckerberg Donor Advised Fund (DAF)… We are very excited to welcome you as part of the Rare As One Network, and we look forward to supporting and learning with you as you work to develop and strengthen a patient-driven collaborative research network in your disease area.”
 
We had to hold our tongues and sit on our hands until the public announcement on February 3, 2020. Much excitement, including press coverage, followed, and then it came time for us to get to work. The purpose of the grant is not to directly fund research but, rather, to enable us to grow and refine our organization, helping provide us with knowledge, tools, and resources to ensure sustainability so that we have the capacity to continue to fund the research until everyone with Usher 1F is cured.
 
Read more about what our CZI grant has helped us achieve
 

Scientific Advisory Council

Almost seven years ago, Usher 1F Collaborative was founded with the explicit goal of finding an effective treatment to save or restore the vision of those with Usher Syndrome type 1F. Since then, there has been prolific scientific research thanks to the almost $5 million contributed by generous donors.
           
Step by step, progress has been steadily advancing in our laboratories. Just one year after being established, over six figures of funding had already been delivered to a lab at the University of Oregon. Within the first two years of our existence, we had not one, but two animal models; a zebrafish and a mouse. In our quest to develop and test a treatment, we have seen great strides since the development began of gene, drug, and stem cell therapies in 2016. Just this year, zebrafish vision gene therapy testing began, and significant progress with gene therapies has been made, including restoring hearing in a mouse model.
           
None of this would have been possible without the researchers who devote their work to our mission. Recognizing the integral role that scientists play in the hunt for a treatment, Usher 1F Collaborative has decided to form a Scientific Advisory Council. This body of three will provide strategic guidance and will advise on timing and implementation of proposals. We are thrilled to announce that our Scientific Advisory Council will include the following members: David R. Liu PhD, Aravinda Chakravarti PhD, and Richard Cummings, PhD.

Please read more about these world-renowned scientists who are donating their time and knowledge to our work for a cure.