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US 10,799,591 B2Granted
Branched oligonucleotides
AssigneeUniversity of Massachusetts
InventorsAnastasia Khvorova, Matthew Hassler, Julia Alterman, Bruno Miguel da Cruz Godinho
Life of this patent
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Abstract
Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.
Claims16
A method of delivering double-stranded nucleic acids to one or both of the brain and spinal cord of a patient, comprising administering to the patient a branched oligonucleotide compound comprising two siRNA duplexes connected to one another by a linker,
- wherein each siRNA duplex comprises a sense strand of at least 15 contiguous nucleotides and an antisense strand of at least 15 contiguous nucleotides having complementarity to the sense strand,
- wherein each sense strand and each antisense strand has a 5′ end and a 3′ end, and
- wherein the branched oligonucleotide compound is administered to the patient by intrastriatal (IS) injection, intrathecal (IT) injection, or intracerebroventricular (ICV) injection, thereby delivering the double-stranded nucleic acids to the brain and/or spinal cord of the patient.
Dependencies are inferred from the claim text.