22 results about "Stargardt disease" patented technology

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.

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.

Disclosed is a method of treating a patient with Stargardt Disease. Also disclosed is a method of delaying the progression of vision loss in a patient with Stargardt Disease. The methods comprise administering to the patient from 10 mg to 35 mg per day of gildeuretinol acetate.

Disclosed herein are compositions and methods for treating ocular diseases in mammals, and particularly complications associated with fundus yellow spot disease. The present disclosure provides an AAV-based dual vector system that promotes expression of a full-length protein having a coding sequence that exceeds the polynucleotide packaging capacity of a single AAV vector. In particular, described herein are methods and compositions relating to the expression of full length ABCA4 using an AAV-based dual vector system.

The present invention relates to the field of medicine. In particular, it relates to a gene editing system that may be used in the treatment, prevention and / or delay of ABCA4-related diseases such as Stargardt disease.

The invention relates to the fields of medicine and biotechnology. In particular, it relates to novel antisense oligonucleotides (AONs) that may be used in the treatment, prevention and / or delay of Stargardt disease and / or ABCA4-associated eye disease. More in particular, the invention relates to AONs that are used in inhibiting or blocking exon 39 skipping in the human ABCA4 pre-mRNA.

The present disclosure relates generally to compositions and methods of preventing, reducing risk of developing, or treating an inherited retinal disease (IRD) (e.g., retinitis pigmentosa, choroideremia, Stargardt disease, cone-rod dystrophy, leber congenital amaurosis), X-linked RP, and Usher Syndrome or retinal detachment.

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.

Disclosed is a method of delaying the onset of Stargardt disease symptoms in an asymptomatic Stargardt disease patient. Also disclosed is a method of delaying the onset of vision loss in an asymptomatic Stargardt disease patient. Also disclosed is a method of treating a Stargardt disease patient to delay the onset of vision loss in the Stargardt disease patient, wherein treatment is initiated prior to the patient experiencing vision loss resulting from the Stargardt disease. The methods comprise administering to the patient an effective amount of gildeuretinol acetate.

The present disclosure describes methods of using lipid nanoparticle (LNP) compositions for treating ocular- related disorders or diseases. In particular, methods are provided for using LNP compositions in gene therapy for targeting an ATP binding cassette subfamily A member 4 (ABCA4) or a functional fragment thereof, in a subject, thereby treating or mitigating Inherited Macular Degenerations including a Stargardt disease or other diseases that involve retinal degeneration.

Compositions and methods for editing a pathogenic ATP-binding cassette, subfamily A, member 4 (ABCA4) polypeptide-encoding gene using an adenosine deaminase base editor to treat a congenital eye disorder, such as Stargardt disease. In various embodiments, the disclosure provides methods for altering a nucleobase (e.g., c.4139T) in an ABCA4 gene codon 1380 encoding a pathogenic leucine so that the codon is altered to encode a proline. In some embodiments, the disclosure provides methods for altering a pathogenic c.5714+5A intronic nucleotide of anABCA4 gene so that the nucleotide becomes c.5714+5G.

Provided herein are methods, systems, and compositions for ARMMs-mediated treatment of ABCA4 retinopathies such as Stargardt disease (STGD1) and Fundus flavimaculatus (FFM).

The present invention relates to RNA editing oligonucleotides (EONs) capable of effecting specific editing of a target nucleotide (adenosine) in a target RNA molecule in a eukaryotic cell, wherein said oligonucleotides are for use in the treatment of Stargardt's disease, more preferably for deamination of a target adenosine present in ABCA4 pre-mRNA or ABCA4 mRNA.

The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.

The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.

The disclosure provides dual AAV vector compositions and systems for reconstitution of RNA molecules, including methods for using these molecules. For example, such molecules can be used to deliver a coding sequence for a ABCA4 gene over two AAV vectors, resulting in reconstitution of the full-length ABCA4 protein in a cell. Such methods can be used to deliver therapeutic ABCA4 protein to treat Stargardt Disease.

The present invention relates to the field of medicine. In particular, it relates to a gene editing system that may be used in the treatment, prevention and / or delay of ABCA4-related diseases such as Stargardt disease.

Provided herein are nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs); RNA exon editing molecules) capable of correcting mutations in the ABCA4 gene. Such molecules are useful in the treatment of disorders such as ABCA4-associated retinal dystrophies (e.g., Stargardt Disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splicing molecules described herein to correct mutations in ABCA4, thereby treating disorders associated with mutations in ABCA4 and use of the nucleic acid trans-splicing molecules described herein for treating disorders associated with mutations in ABCA4 and in the preparation of medicaments for the treatment of disorders associated with mutations in ABCA4.

The present disclosure describes methods of using lipid nanoparticle (LNP) compositions for treating ocular- related disorders or diseases. In particular, methods are provided for using LNP compositions in gene therapy for targeting an ATP binding cassette subfamily A member 4 (ABCA4) or a functional fragment thereof, in a subject, thereby treating or mitigating Inherited Macular Degenerations including a Stargardt disease or other diseases that involve retinal degeneration.

Disclosed is a method of delaying the onset of Stargardt disease symptoms in an asymptomatic Stargardt disease patient. Also disclosed is a method of delaying the onset of vision loss in an asymptomatic Stargardt disease patient. Also disclosed is a method of treating a Stargardt disease patient to delay the onset of vision loss in the Stargardt disease patient, wherein treatment is initiated prior to the patient experiencing vision loss resulting from the Stargardt disease. The methods comprise administering to the patient an effective amount of gildeuretinol acetate.

Provided herein are nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs); RNA exon editing molecules) capable of correcting mutations in the ABCA4 gene. Such molecules are useful in the treatment of disorders such as ABCA4-associated retinal dystrophies (e.g., Stargardt Disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splicing molecules described herein to correct mutations in ABCA4, thereby treating disorders associated with mutations in ABCA4 and use of the nucleic acid trans-splicing molecules described herein for treating disorders associated with mutations in ABCA4 and in the preparation of medicaments for the treatment of disorders associated with mutations in ABCA4.

Aspects of the disclosure relate to compositions and methods useful for delivering minigenes to a subject. Accordingly, the disclosure is based, in part, on isolated nucleic acids and gene therapy vectors, such as viral (e.g., rAAV) vectors, comprising one or more gene fragments encoding a therapeutic gene product, such as a protein or peptide (e.g., a minigene). In some embodiments, the disclosure relates to gene therapy vectors encoding a ABCA4 protein (e.g., the gene product of ABCA4 gene) or a portion thereof. In some embodiments, compositions described by the disclosure are useful for treating diseases associated with mutations in the ABCA4 gene, for example Stargardt disease.