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Repair of Aberrant Splicing in Growth Hormone Receptor by Antisense Oligonucleotides Targeting the Splice Sites of a Pseudoexon
2010
Endocrine reviews
Context: The GH receptor (GHR) pseudoexon 6⌿ defect is a frequent cause of GH insensitivity (GHI) resulting from a non-functioning GH receptor (GHR). It results in a broad range of phenotypes and may also be present in patients diagnosed as idiopathic short stature. Objective: Our objective was to correct aberrant GHR splicing and inclusion of 6⌿ using exonskipping antisense oligonucleotides (ASOs). Design and Setting: Three ASOs binding the 5Ј (ASO-5), 3Ј (ASO-3), and branch site (ASO-Br) of
doi:10.1210/edrv.31.3.9984
fatcat:3ygd5jqakzaqnjg6zlg5nwaym4