USE OF T-LOOP IN TRANS-SPLICING POLYNUCLEOTIDES

摘要

Provided herein are compositions and methods comprising a pre-therapeutic molecule (PTM) for the production of a therapeutic molecule in cells. The therapeutic molecule can be produced in a host, such as an animal. The cell can be deficient for a protein or microRNA, produce a defective protein or microRNA, or overproduce a miRNA. The PTM disclosed herein can be used for targeted delivery to the cell, resulting in the correction of a cellular defect.