Search Results - genetic+disorder

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Methods and compositions for treating ataxia telangiectasia

Methods and compositions for treating ataxia telangiectasia Background Ataxia telangiectasia (A-T) is an incurable autosomal recessive disease that occurs in 1 in 40,000 to 100,000 people worldwide. It is caused by mutations in the Ataxia Telangiectasia Mutated (ATM) gene. A-T is characterized by neurodegeneration in the central nervous system, immune...

Published: 6/26/2025 | Updated: 5/30/2023 | Inventor(s): Timothy Yu, Jinkuk Kim

Keywords(s): CNS, Gene Therapy, Genetic Disorder, Rare Disease

Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > ASOs, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Neurology

Oligonucleotide approach to treat exon skipping diseases

Oligonucleotide approach to treat exon skipping diseases Background Almost half of mammalian genomes contain repeat DNA sequences known as transposable elements (TE) or transposons. Many transposons have the ability to mobilize and change location in the genome, yet their role in human health and disease is not well defined. RNA transposons (i.e.,...

Published: 3/20/2026 | Updated: 5/30/2023 | Inventor(s): Timothy Yu, Alice Lee

Keywords(s): CNS, Gene Therapy, Genetic Disorder, Rare Disease