The scientific and ethical community is continuing the debate on whether the CRISPR gene editing tool is ready to be used safely to edit embryos to be implanted, in response to Unverified statements made Sunday that a successful birth of twins modified by a gene would be possible. modified pregnancy.
What's New: The chief scientist of the controversial allegation, He Jiankui, presented some of his data at an international conference in Hong Kong. However, David Baltimore, chairman of the organizing committee, said the research did not meet what was considered the basic criteria for this type of experiment to begin.
"This was not the case, and he would still be considered irresponsible."
– David Baltimore stated that he personally expressed himself at the conference.
Background: Although the science behind gene editing has flourished over the past decade, especially With the invention of the CRISPR tool more accurate there is About 6 years, most researchers say that the technology is far from ready to be used on human embryos and that this could have repercussions not only on the child, but on subsequent generations.
is seriously concerned about the use of CRISPR for editing genes in adults with inherited diseases, including an untargeted edition that may cause cancer and / or large deletions and a rearrangement of DNA.
Worr are still far more important than the edition of embryos, as this adds many unknowns about altering the basic germinal DNA that are pbaded on to future generations, explains Eric Topol, founder and director of the Scripps Research Translational Institute,
- . In addition to off-target editing or large deletions, another area of concern is mosaicism, where there may be a mix of edited and unedited genes that allow the disease or targeted health problem to continue.
Headlines: International Outrage has increased in recent days since the Chinese team led by He told AP that she had used CRISPR-Cas9 to edit CCR5, a gene known for play a role in HIV infection by cells, in embryos of 7 couples where the fathers were seropositive during fertility treatments.
"It's terribly unfortunate because we're far from ready for it."
– Eric Topol
As a result of claims not yet verified – although he claims that they have been submitted to a peer-reviewed scientific journal – some scientists, including including co-inventors of CRISPR, call for a moratorium on the implantation of gene-modified embryos until further research can be conducted to determine safety and efficacy. 39; efficiency.
Details of the Claims
per He makes a presentation at the conference and his speech on YouTube:
- During IVF Fertilization Procedures, Right After Fertilization of Embryos with Sperm , the team published the CCR5 gene of each single cell embryo with CRISPR-Cas9 and "removed the door through which HIV enters to infect people."
- They developed the embryos a few days outside the uterus and controlled the development of the embryo with sequencing of the entire genome. He added that they had discovered that an embryo had a potential off target (but that the location was far from the relevant genes) and that a mosaic embryo had changed only one of the two strands of DNA.
- Despite these problems, the parents decided to implement. both embryos, after giving informed consent according to He. They implanted the 2 embryos, monitored the pregnancy closely with ultrasound and blood tests and indicated that the pregnancy was progressing normally.
- The couple gave birth to "healthy" twins a few weeks ago, according to He, who added that there could be a second early pregnancy of one of the other couples.
- According to him, the purpose of the review is to prevent children from becoming infected with HIV in the future, and not in the womb where the risk is already minimal.
But, but, but: The scientific colleagues of the conference expressed serious concerns …
- Why target this gene while there are other conditions for which no protection alternative is available against HIV. Topol claims that choosing CCR5 as a gene to modify is "ridiculous".
- How was the informed consent process conducted and how could results be verified if patients remain anonymous?
- What will be the life of the two children
- How was this financed and if both of its companies were part of it (He says no)
- Why there was such a lack of transparency in the development and conduct of the trial.
The process was transparent and we only learned about it when the children were born. There has been a failure of self-regulation on the part of the scientific community. "
– David Baltimore, also Nobel Laureate [19659002] On the other side: For him, he considers gene editing as" another progress of IVF "and should be used by a small group of chosen parents who do not seek so-called "designer babies", but rather to prevent what could be a painful and painful life for the child.
"A gene surgery that could save a child from a deadly genetic disease such as cystic fibrosis or a deadly infection like HIV does not just give that little girl or boy an equal chance to lead a healthy life. We heal a whole family. "
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