Disclosed is a method of treating a subject who has a neurological disease. The neurological disease may be associated with altered C9ORF72 protein activity. In one aspect, the method includes a step of administering an effective dose of a PIKFYVE antisense or inhibitory nucleic acid to a subject in need thereof, thereby rescuing the defects associated with altered C9ORF72 protein activity and/or inhibiting the expression of PIKFYVE gene.

The present disclosure provides an anti-VEGF aptamer, an agent or composition comprising the anti-VEGF aptamer, as well as uses thereof.

The present disclosure provides a trinucleotide comprising the formula below or an oligomeric compound comprising the formula below:

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The present disclosure provides a trinucleotide comprising the formula below or an oligomeric compound comprising the formula below:

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Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

The present invention relates to nucleic acid molecules that are complementary to both PAP associated domain containing 5 (PAPD5) and PAP associated domain containing 7 (PAPD7), leading to inhibition of the expression of both PAPD5 and PAPD7 when using a single nucleic acid molecule. The invention also provides for PAPD5 and PAPD7 specific nucleic acid molecules for use in treating and/or preventing a HBV infection, in particular a chronic HBV infection. Also comprised in the present invention is a pharmaceutical composition for use in the treatment and/or prevention of a HBV infection.

The present invention relates to oligonucleotides that are complementary to and modulate the expression of TMEM106B. The present invention further relates to conjugates of the oligonucleotide and pharmaceutical compositions and methods for treatment of neurological disorders using the oligonucleotide.

The present invention relates to the use of an antisense oligonucleotide targeting LINC00518 for the treatment of melanoma.

The present discloses relates to a composition, a kit or a method using an eIF4E inhibitor for diagnosis or treatment of a condition associated with increased activity of eIF4E.

Compositions related to spherical nucleic acids (SNAs) with antisense oligonucleotides and methods of treatment of diseases and disorders are disclosed therein. In particular, the antisense oligonucleotides are targeted to a region in a pre-mRNA of interest to regulate pre-mRNA splicing.