Compounds and methods for modulating PLP1
AssigneeIonis Pharmaceuticals, Inc.
InventorSusan M. Freier
Life of this patent
Estimating the term…
Abstract
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PLP1 RNA in a cell or subject, and in certain instances reducing the amount of proteolipid protein 1 in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include hypotonia, nystagmus, optic atrophy, respiratory distress, motor delays, cognitive dysfunction, speech dysfunction, spasticity, ataxia, seizures, choreiform movements, and death. Such leukodystrophies include Pelizaeus-Merzbacher disease.
Claims32
A modified oligonucleotide according to the following chemical structure:
- or a salt thereof.
A modified oligonucleotide according to the following chemical structure:
An oligomeric compound comprising a modified oligonucleotide according to the following chemical notation:
- (SEQ ID NO:
- 2163)
- TesGeoTeoAeoGeoTdsAdsmCdsAdsAdsAdsTdsmCdsTdsTdsTeo
- mCeomCesTesTe,
- wherein:
- A=an adenine nucleobase,
- mC=a 5-methylcytosine nucleobase,
- G=a guanine nucleobase,
- T=a thymine nucleobase,
- e=a 2′-MOE sugar moiety,
- d=a 2′-β-D-deoxyribosyl sugar moiety,
- s=a phosphorothioate internucleoside linkage, and
- o=a phosphodiester internucleoside linkage.
Dependencies are inferred from the claim text.