Usher Syndrome Ireland
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    • Home
    • Information
      • What is Usher Syndrome?
      • Usher Syndrome - Genetics
      • Adults - Newly Diagnosed?
      • Adults - USH Community
      • Newly Diagnosed Child?
      • Telling Your Child
      • Parents - USH Community
      • USH Connect
    • HCPs
      • Information for HCPs
      • Overview - Usher Syndrome
      • Create Accessibility
      • Talks/Presentations
    • Research
      • Usher Syndrome Research
      • Understanding USHResearch
      • USH Patient Registries
      • USH Research News
    • More Info & Resources
      • Usher Syndrome Leaflets
      • Yellow Sticker Campaign
      • Life with USH - Blog
      • Help & Resources
      • Archived Newsletters
    • About Us
      • About Us
      • Meet Our Team
      • Contact Us
    • Support Us
      • Support Us
      • Donate Today
    • Q&A with Jeremy
  • Home
  • Information
    • What is Usher Syndrome?
    • Usher Syndrome - Genetics
    • Adults - Newly Diagnosed?
    • Adults - USH Community
    • Newly Diagnosed Child?
    • Telling Your Child
    • Parents - USH Community
    • USH Connect
  • HCPs
    • Information for HCPs
    • Overview - Usher Syndrome
    • Create Accessibility
    • Talks/Presentations
  • Research
    • Usher Syndrome Research
    • Understanding USHResearch
    • USH Patient Registries
    • USH Research News
  • More Info & Resources
    • Usher Syndrome Leaflets
    • Yellow Sticker Campaign
    • Life with USH - Blog
    • Help & Resources
    • Archived Newsletters
  • About Us
    • About Us
    • Meet Our Team
    • Contact Us
  • Support Us
    • Support Us
    • Donate Today
  • Q&A with Jeremy

Eye on the Research

Gene replacement

A list of gene replacement studies currently underway that may potentially benefit patients with Usher syndrome. Please refer to the link under each trial for more information on that trial. Please be aware, all information provided are for informational purposes only, and are in no way endorsed or approved by Usher Syndrome Ireland. 


Pre-Clinical Development Stage


  • Atsena Therapeutics is making rapid advancements in evaluating a dual-AAV vector-based gene therapy that could potentially prevent blindness from MYO7A-associated Usher Syndrome 1B (USH1B). More information can be found here: https://atsenatx.com/programs/ush1b/ 


Early Research Stage


  • Standard USH2A genes are too large to fit into a single AAV used in gene therapy to replace a faulty gene with a functional copy. Iveric Bio is currently in early research stage aiming to develop a smaller USH2A construct (minigene) to fit into an AAV and transcribe a functional protein. More information can be found here: https://ivericbio.com/why-iveric/

 << Click here to return to  'Understanding USH Research'  


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+353 (89) 262 4696 

(Mon-Fri 10am-4pm)


Address

15 Windsor Terrace, 

Dun Laoghaire, Co. Dublin, 

A96 RF29


Reg. Number: 688621 

Reg Charity Number: 20206288


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