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Exclusive Excerpts from Unseen Research on China's CRISPR Babies

He Jiankui, the Chinese biophysicist, authored an unpublished manuscript titled "Birth of Twins After Genome Editing for HIV Resistance," which describes the creation of the first gene-edited babies.
The manuscript is 4,699 words long and was edited by He in late November 2018. It was considered by two prestigious journals, Nature and JAMA, but remains unpublished.
The manuscript claims "success" in using a "novel therapy" to render the edited twin girls resistant to HIV, but makes little attempt to prove it.
The researchers' claim that gene-editing babies could save millions of people from HIV infection is called "preposterous" and "ludicrous" by experts.
The manuscript does not include the names of the fertility doctors who treated the patients or the obstetrician who delivered the babies, leaving unclear whether they understood they were helping to create the first gene-edited babies.
The research has been criticized for ignoring ethical and scientific norms and moving forward with creating living human beings before fully understanding the effects of the edits they had made.
The purpose of the editing was to give the children immunity to HIV later in life, not to prevent them from catching HIV from their father, who was infected.
The couple who agreed to the experiment may have done so to access fertility treatment, as the husband was HIV-positive, and HIV-positive patients have no access to treatment for infertility in China.
The gene edits weren't the same as the mutations that confer natural HIV resistance, and the researchers never checked to see if the new mutations would lead to HIV resistance.
The manuscript fails to provide clear, immediate medical benefits to either the parents or the children.
Experts call into question the validity of the manuscript and the researchers' claims, given the lack of independent evidence and the circumstances surrounding the research.
The research remains controversial and raises important questions about the ability to change human heredity using technology.Exclusive Excerpts from China's CRISPR Babies Research
A Chinese scientist named He Jiankui claimed to have edited the genes of twin girls in an attempt to make them resistant to HIV.
The research manuscript related to this experiment reveals that the editing was incomplete, and only one of the embryos had edits to both copies of the CCR5 gene, giving partial HIV resistance at best.
The manuscript shows that the researchers were aware of the possibility of unwanted CRISPR edits and only found one such mutation, but the search was incomplete.
The doctors who treated the couple may not have been aware of the gene-editing experiment, and there are allegations that He Jiankui's team tricked doctors by switching blood samples.
The manuscript included a false date for the birth of the twins, which may have been an attempt to protect their anonymity.
It is not clear if there was a proper ethics review for the experiment, and the research plan was only registered after the twins were born.
The researchers did not test whether the HIV immunity worked before creating living human beings, which was a violation of elementary norms of ethics and research.
He Jiankui thanked several people for their advice and feedback on the manuscript, including Mark Dewitt, who warned against the project, and William Hurlbut, who gave ethics advice to He without knowing that children had been created.
Twink Allen, an equine reproduction specialist in the UK, and Jin Zhang, who is head of New Hope Fertility Center in New York, were also thanked, and Zhang was reportedly planning to open a medical tourism business for gene-edited babies with He.
The manuscript did not provide information about who funded the project or what financial interests the authors have in the outcome, and there was no section detailing each author's scientific contribution.
Data attached to the paper shows that the embryos were "mosaic," meaning different cells in the embryo were edited differently, which was ignored by the researchers.
The nature of Michael Deem's role in the project, particularly any hands-on involvement with the patients, could determine penalties that Deem or his university could face, and Rice University says its investigation is ongoing.Unseen Original Research on China's CRISPR Babies
The original research by He Jiankui on the CRISPR-edited babies in China has been obtained and reviewed by MIT Technology Review.
He Jiankui used a powerful gene-editing tool called CRISPR to edit the genes of twin girls in an attempt to make them resistant to HIV.
The research shows that the babies have unexpected changes in their DNA, including a deletion that might shorten their lifespan.
The deleted gene is called CCR5, which plays a role in the immune system and is known to be important for the survival of humans.
The research also shows that the girls are genetic mosaics, which means their bodies could be composed of cells edited in different ways, or not edited at all.
Only some of their cells might have the HIV-resistant gene edit, and some might have undetected "off-target" edits, which could cause health problems.
He Jiankui knew about the problem of mosaicism from his experiments on animal embryos, but it is unclear why he chose to proceed with human embryos despite this issue.
The research manuscript does not resolve this mystery and does not acknowledge that the data seem to show mosaicism or that it’s a problem.
The research has been criticized by experts in the field for being unethical, unsafe, and scientifically unsound.
The Chinese government has denounced He Jiankui's work and has sentenced him to three years in prison for illegal medical practices.
The case has raised concerns about the regulation of gene-editing technology and the need for international guidelines to prevent similar incidents from happening in the future.
CRISPR technology has the potential to revolutionize medicine, but it also raises ethical and safety issues that need to be addressed before it can be widely used in humans.