USHER SYNDROME COALITION
Usher Syndrome Coalition |usher-syndrome.org | Westford, MA | 800.946.9203 Mission The Usher Syndrome Coalition’s mission is to raise...
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- Jul 23, 2022
Potential of endogenous regenerative technology for in situ regenerative medicine
Endogenous regenerative technology involves the use of a patient's biologically active proteins, growth factors and biomaterial scaffolds.
- Jun 17, 2022
A new hope for a therapy against retinitis pigmentosa
June 17, 2022 | Université de Genève Journal Reference Olivier Mercey, Corinne Kostic, Eloïse Bertiaux, Alexia Giroud, Yashar Sadian,...
- Apr 30, 2022
Retinal Phenotype of Patients with CLRN1-Associated Usher 3A Syndrome in French Light4Deaf Cohort
Biallelic variants in CLRN1 are responsible for Usher syndrome 3A and non-syndromic rod–cone dystrophy (RCD).
- Mar 18, 2022
Antabuse may help revive vision in people with progressive blinding disorders
UC Berkeley researchers have found that a drug once widely used to wean alcoholics off drinking may help improve sight in mice with RD.
- Jan 31, 2022
MalaCards: The human disease database
MalaCards is an integrated database of human maladies and annotations, modeled on the architecture and richness of the popular GeneCards.
- Jan 31, 2022
ProQR seeking Clinical Trial Participants
Usher Syndrome Coalition announces partnership with ProQR to support clinical trial enrollment for a potential therapy for USH2A.
- Jan 24, 2022
RPGR and X-linked Retinitis Pigmentosa
Among the most common genetic causes of IRDs are mutations in the RPGR gene located on the X-chromosome.
- Jan 19, 2022
Eluminex Biosciences Acquires Zuretinol Acetate from Retinagenix Holdings
Eluminex announced today it acquired rights for a novel oral therapy to treat LCA or RP caused mutations of the RPE65 or LRAT gene.
- Jan 1, 2022
Retinitis Pigmentosa Pathway
Many genes have been identified for the inherited and highly heterogeneous disorders - some are arRP, adRP and X-linked.
- Dec 21, 2021
First patients dosed in phase 2/3 gene therapy trial for retinitis pigmentosa
The first patients have been dosed in the phase 2/3 Sirius and Celeste clinical trials investigating QR-421a in patients with USH2A.
- Sep 16, 2021
Coave Therapeutics and Théa Open Innovation sign exclusive agreement
Coave Therapeutics enters into an exclusive licensing, co-development, and commercialization agreement with Théa Pharma.
- Aug 16, 2021
New Treatments for Retinitis Pigmentosa
Existing treatments only help a fraction of the estimated 100,000 Americans with this condition. But advances in gene therapy may soon help.
- Jul 28, 2021
Pre-mRNA Processing Factors and Retinitis Pigmentosa: RNA Splicing and Beyond
Mutations in pre-mRNA processing factors (PRPF3, 4, 6, 8, 31, SNRNP200, and RP9) have been linked to 15–20% of autosomal dominant RP cases.
- Jul 13, 2021
CRISPR/Cas9 Gene Editing
X-linked RP caused by RPGR mutations, an attractive target for gene therapy because of its clinical severity and large number of patients.
- Jul 5, 2021
The Lrat−/− Rat: CRISPR/Cas9 Construction and Phenotyping of a New Animal Model for RP
Researchers developed and phenotyped a pigmented knockout rat model for lecithin retinol acyltransferase (LRAT) using CRISPR/Cas9.
- Jun 28, 2021
Broadening INPP5E phenotypic spectrum:
Here, we report 16 non-syndromic IRD patients and two cases with an IRD and some JBTS clinical features with pathogenic variants in INPP5E.
- May 24, 2021
Clinical Trial News: Positive results in Usher syndrome and RP clinical study
ProQR news – Positive results achieved in our ongoing Usher syndrome and retinitis pigmentosa research.
- May 10, 2021
2021 Spring Fundraiser - April 24th
Before sharing a summary of our inaugural fundraiser, we again want to thank all who joined us on Saturday, April 24th. It was such a...
- Apr 26, 2021
An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis
This review provides an overview of retinal gene therapy development by summarizing significant contributions and important clinical trials.
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