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Gene Editing to Correct Genetic Problems in Human Embryos is a Step Closer to Reality

CRISPR” (pronounced “crisper”) stands for Clustered Regularly Interspaced Short Palindromic Repeats, and researchers have developed a new version, CRISPR-Cas9, to edit the base pairs of genes. Yesterday, in the Journal " Nature Medicine" a team of researchers announced that they had successfully edited genes in mouse fetuses to correct a condition known as hereditary tyrosinemia type 1, a potentially fatal genetic disease.

The use of CRISPR on humans is still a few years away, but researchers are excited about its potential to cure a wide variety of genetic diseases.

The Guardian: "Robin Lovell-Badge, head of the division of stem cell biology and developmental genetics at the Francis Crick Institute in London, welcomed the study."

“These are important proof-of-principle experiments that suggest that genetically inherited liver diseases might be avoided by correcting a defect before the effects of the mutation will have compromised liver function in the newborn child,” he said."

"Lovell-Badge also said the base editing methods “are particularly exciting, because up to 50% of inherited mono-genetic diseases (where the defect is due to a mutation in a single gene) are due to single base-pair substitutions.”

Here is an earlier article we wrote about CRISPR and how the technology will change our world in ways we can't even imagine yet, and it's already started.

#science #crispr

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