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VISIONS 2015 – Dr. Shannon Boye Receives FFB Award for Excellence in Gene-Therapy Research

Shannon Boye accepting the awardWhen it comes to developing innovative gene therapies for retinal diseases, few researchers are doing more than Shannon Boye, Ph.D., and her laboratory staff of 10 at the University of Florida. In addition to advancing a gene therapy for Leber congenital amaurosis (LCA) caused by GUCY2D mutations toward a human study, Dr. Boye and her team are enhancing gene-delivery systems by making them safer to administer and able to carry larger payloads than current systems.

In recognition of her pivotal role in moving the gene-therapy field forward, Dr. Boye received the Foundation’s Board of Director’s Award, which was presented at VISIONS 2015, FFB’s annual conference, for achievements in retinal research.

“Not only is Dr. Boye a top-notch retinal investigator; she is thoughtful, articulate and, as VISIONS attendees are learning in her Gene Therapy 101 session, she knows how to communicate the complicated science in terms that everyone can understand,” says Stephen Rose, Ph.D., the Foundation’s chief research officer.

Dr. Boye’s emerging LCA gene therapy is particularly compelling because GUCY2D mutations severely affect the cones of young children. Cones are the retinal cells responsible for reading, recognizing faces, perceiving colors and seeing in lighted conditions.

She has achieved success with the gene therapy in mouse models of LCA, and is now determining the optimal viral gene-delivery system for a planned human study. She’s partnering with the pharmaceutical company Genzyme to launch the clinical trial.

Dr. Boye is also working on a technique for injecting gene therapies into the middle of the eye, which is filled with pliable vitreous gel. The approach is less likely to cause damage than subretinal injections, which are commonly used but more invasive, and put the retina, already fragile from disease, at risk of further damage.

To address the challenge of getting large genes like USH2A and CEP290 into cells of the retina, Dr. Boye is developing what’s known as a dual-vector delivery system, which essentially doubles the capacity of current systems.

Dr. Boye is one of the youngest investigators ever to receive the Board of Director’s Award, which is given annually at the conference. “Young researchers are beginning their careers with more powerful technologies and more scientific knowledge than established scientists like we had when starting out,” says Dr. Rose. “They have an opportunity to advance the science much faster and further. They are the lifeblood of the future of research, and moving treatments into human studies.”

The Foundation established the Alan Laties Career Development Program to foster the advancement of young, retinal-research talent. It’s given out more than 100 awards.


2 Responses to 'VISIONS 2015 – Dr. Shannon Boye Receives FFB Award for Excellence in Gene-Therapy Research'

  1. Art wade says:

    I have three adult children,22,20 and 18 with Ushers syndrome. Is there somewhere you can direct me to for treatments or clinical trials that may help?

    • EyeOnTheCure says:

      Usher syndrome is the most common condition that affects both hearing and vision. There are three general types of Usher syndrome. People with Usher syndrome type 1 (USH1) are born completely deaf and experience problems with balance. The first signs of RP — night blindness and loss of peripheral vision — usually appear in early adolescence. In Usher syndrome type 2 (USH2), newborns have moderate to severe hearing impairment. Symptoms of RP typically start shortly after adolescence. Visual problems progress less rapidly than in Usher type 1 and hearing loss usually remains stable. A rarer third type of Usher syndrome (USH3) was documented in 1995. Children with USH3 are usually born with good or only mild impairment of hearing. Their hearing and vision loss is progressive, starting around puberty. Balance may be affected. For more detailed information on Usher syndrome, please see the following web link: http://www.blindness.org/eye-conditions/usher-syndrome
      The Foundation Fighting Blindness is currently supporting a gene therapy trial for Usher type 1B. For more information on this clinical trial, please see the following link:

      http://www.blindness.org/foundation-news/usher-syndrome-gene-therapy-study-begins-oregon

      To participate in any gene therapy trial, you must first positively identify the gene mutation. For more information on genetic testing, please see the following link:

      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf

      You may also want to consider enrolling in “My Retina Tracker”, a free registry that can help people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

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