Deadly genetic disease prevented before birth in zebrafish
March 20, 2008 | Source: KurzweilAI
Washington University School of Medicine researchers corrected a genetic mutation by injecting a customized “genetic patch” into early-stage fish embryos, allowing the embryos to develop and hatch normally.
The research could lead to prevention of one-fifth of birth defects in humans caused by genetic mutations, according to the researchers.
They used a zebrafish model of Menkes disease, a rare, inherited disorder of copper metabolism. Most children with Menkes die before age 10, and treatment with copper is largely ineffective.
Menkes is caused by splicing defects, or an interruption in genetic code. The researchers designed a specific therapy to correct each mutation with morpholinos, synthetic molecules that modify gene expression. The morpholinos patched over the defect so the gene could generate its normal product.
