What are different approaches to gene therapy? Discuss the success and failure of each.
What are different approaches to gene therapy? Discuss the success and failure of each.
Answered
Gene Addition: Involves adding a functional gene to replace a defective one. Successes include treatments for certain genetic disorders like SCID (severe combined immunodeficiency). Failures can occur due to insertional mutagenesis or inadequate gene expression.
Gene Editing: Uses techniques like CRISPR/Cas9 to directly modify the genome. Successes include treating genetic diseases by correcting mutations. Challenges include off-target effects and ethical concerns.
Gene Silencing: Involves turning off or reducing the expression of a specific gene. RNA interference (RNAi) is an example. Successes include treating diseases caused by overactive genes. Failures may include insufficient silencing or unintended consequences on gene expression.
Gene Replacement: Replaces a defective gene with a normal copy. Successes include the treatment of certain inherited diseases. Challenges include ensuring proper regulation and integration of the new gene.
