CHOP Researchers Develop Versatile and Low-Cost Technology for Targeted Long-read RNA Sequencing

In a development that could accelerate the discovery of new diagnostics and treatments, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a versatile and low-cost technology for targeted sequencing of full-length RNA molecules.

New Computational Tool Identifies Novel Targets for Cancer Immunotherapy

Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of California, Los Angeles (UCLA) have developed a computational platform capable of discovering tumor antigens derived from alternative RNA splicing, expanding the pool of cancer immunotherapy targets. The tool, called “Isoform peptides from RNA splicing for Immunotherapy target Screening” (IRIS), was described in a paper published today in the Proceedings of the National Academy of Sciences.

Children’s Hospital of Philadelphia Researchers Develop “Dimmer Switch” to Help Control Gene Therapy

In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a “dimmer switch” system that can control levels of proteins expressed from gene therapy vectors. The system is based on alternative RNA splicing using an orally available small molecule and works effectively in tissues throughout the body, including the brain. The first research regarding this innovation was published today in the journal Nature.