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3 results about "Stargardt disease" patented technology
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Stargardt disease is the most common inherited single-gene retinal disease. It usually has an autosomal recessive inheritance caused by mutations in the ABCA4 gene. Rarely it has an autosomal dominant inheritance due to defects with ELOVL4 or PROM1 genes. It is characterised by macular degeneration that begins in childhood, adolescence or adulthood, resulting in progressive loss of vision.
trans-splicing molecules
ActiveCN112449605BLeber congenital amaurosisTrans-splicing
The present invention is characterized by nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 or CEP290 genes. Such molecules can be used to treat conditions associated with ABCA4 mutations, such as Stargardt disease (e.g., Stargardt disease 1), and conditions associated with CEP290 mutations, such as Leber congenital amaurosis 10 (LCA 10). The invention also provides methods for using nucleic acid trans-splicing molecules to correct mutations in ABCA4 and CEP290, and for treating conditions associated with mutations in ABCA4 and CEP290, such as Stargardt disease and LCA 10.
Owner:ASCIDIAN THERAPEUTICS INC +1
Methods and Compositions for the ADAR-Mediated Editing of ABCA4
The present invention relates to methods and compositions for editing an ABCA4 polynucleotide, e.g., an ABCA4 polynucleotide comprising a SNP associated with Stargardt Disease, type 1. The invention also relates to methods and compositions for treating or preventing Stargardt Disease, type 1, in a subject.
Owner:KORRO BIO INC
Dual-AAV delivery of ABCA4
Provided herein is a composition or a method for expression of a human ABCA4 protein. Also provided herein is a method of treating Stargardt disease.
Owner:HUIGENE THERAPEUTICS CO LTD +1