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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
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Updated: 5/30/2023
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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
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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
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Updated: 5/30/2023
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Inventor(s): Timothy Yu, Alice Lee
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
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Antisense oligonucleotides that suppress SARS-CoV-2 replication
Antisense Oligonucleotides that Suppress SARS-CoV-2 Replication
Antisense oligonucleotides that suppress SARS-CoV-2 replication by targeting the 5’ untranslated region of SARS-CoV-2
Background
The coronavirus disease 2019 (COVID-19) pandemic has profoundly impacted global human health and is caused by the severe acute respiratory syndrome coronavirus...
Published: 9/26/2025
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Updated: 1/6/2023
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Inventor(s): Hao Wu, Longfei Wang, Setu Vora
Keywords(s):
Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Infectious Disease, Technology Classifications > Disease Area > Infectious Disease > COVID-19, Technology Classifications > Invention Type > Therapeutics > ASOs
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