Reducing intron retention

A method of treating a disease or condition in a subject in need thereof, wherein the subject has impaired production of a functional form of a protein, the method comprising administering to the subject a pharmaceutical composition comprising:

• (i) a polynucleic acid polymer;
• and
• (ii) a pharmaceutically acceptable excipient and/or a delivery vehicle;
• wherein a target cell of the subject has a pool of partially processed mRNA transcripts, which are capable of encoding copies of the functional form of the protein and each of which comprise a retained intron, an exon flanking a 5′ splice site of the retained intron, and an exon flanking 3′ splice site of the retained intron;
• wherein the polynucleic acid polymer contacts the target cell of the subject and increases the production of the functional form of the protein by correcting low efficiency of removal of the retained intron in the partially processed mRNA transcripts by inducing a portion of the pool of the partially processed mRNA transcripts to undergo splicing to remove the retained intron from each of the partially processed mRNA transcripts in the portion, thereby producing fully processed mRNA transcripts and increasing expression of the functional form of the protein
• wherein the polynucleic acid polymer hybridizes to a target region of the retained intron, wherein the target region of the retained intron to which the polynucleic acid polymer hybridizes:
• (a) is an internal region of the retained intron,
• (b) does not comprise a 5′ splice site of the retained intron, and
• (c) does not comprise a 3′ splice site of the retained intron;
• (d) wherein the subject has a genome that comprises:
• (I) a copy of a gene that comprises a set of exons that when properly transcribed into fully processed mRNA encodes the functional form of the protein, and
• (II) a defective copy of the gene, which is incapable of producing the functional form of the protein, thereby resulting in a level of expression that is sufficiently low so as to cause the disease or condition.