Pittsburgh Post-Gazette

Chinese scientists modify DNA of human embroyos

- By Sharon Begley

NEW YORK — Biologists in China reported carrying out the first experiment to alter the DNA of human embryos, igniting an outcry from scientists who warn against altering the human genome in a way that could last for generation­s.

The study from China appeared last weekend in an obscure online journal called Protein & Cell. In an interview published on Wednesday on the news site of the journal Nature, lead author Junjiu Huang of Sun Yat-sen University in Guangzhou said both Nature and Science rejected the paper, partly for ethical reasons.

“There have been persistent rumors” of this kind of research taking place in China, said Edward Lanphier, chief executive of California-based Sangamo BioScience­s Inc and part of a group of scientists who called last month for a global moratorium on such experiment­s.

The controvers­ial technique is called CRISPR/ Cas9, and represents a biological version of a wordproces­sing program's "find and replace” function. Scientists introduce enzymes that first bind to a mutated gene, such as one associated with disease, and then replace or repair it.

At least half a dozen experiment­s have been planned or are underway using CRISPR on human eggs or embryos to correct genetic defects such as those causing cystic fibrosis or the BRCA1 breastcanc­er gene, the Massachuse­tts Institute of Technology magazine, Technology Review, recently reported.

Scientists warn that altering the DNA of human sperm, eggs, or embryos could produce unknown effects on future generation­s, since the changes are passed on to offspring. They distinguis­h this type of so-called germline engineerin­g from that which alters the DNA of non-reproducti­ve cells to repair diseased genes.

“It is too soon to apply these technologi­es to the human germ line, the inherited DNA, in a clinical setting,” said MIT biologist Rudolf Jaenisch, president of the Internatio­nal Society for Stem Cell Research.

Mr. Huang's experiment­s provide evidence of what can go wrong with CRISPR.

His team experiment­ed on 86 one-cell human embryos, they reported, all from fertility clinics and, because of chromosoma­l defects, unable to develop into a baby.

Their target was a gene called HBB, which can cause the blood disease beta-thalassemi­a.

About a dozen embryos did not even survive the genome-editing.

Of the surviving embryos, many showed “offtarget” effects, they reported, meaning genes other than HBB were altered. Other embryos suffered “untoward mutations.” Only a handful of embryos contained the healthy DNA meant to repair the defective HBB genes.

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