The He LabTwo Chinese girls, who we'll call Lulu and Nana to protect their privacy, were born healthy a few weeks ago. Their mother Grace started her pregnancy by regular IVF with one difference: right after sending her husband's sperm into her eggs, an embryologist also sent in CRISPR/Cas9 protein and instructions to perform a gene surgery intended to protect the girls from future HIV infection. The surgery reproduces a natural genetic variation shared by more than 100 million people of primarily European origin that confers strong resistance to initial HIV-1 infection and disease progression. While CRISPR/Cas9 has been studied in human cells and in early clinical trials, gene surgery in embryos intended for pregnancy has not previously been reported. Safety remains a key concern, particularly for unintended changes to the genome. To assess the girls' genomes for safety after the surgery, multiple whole genome and targeted deep sequencing techniques were used before implantation, during the pregnancy, and now after birth. These data indicate the girls' genomes' were changed as intended by the gene surgery, but no off-target editing or large deletions occurred. Further assessments to confirm these findings will be conducted over the next year. We will publish our full data soon.
Email: HeJiankuiLab@gmail.com He Jiankui Lab Website: http://www.sustc-genome.org.cn
About Lulu and Nana: Twin Girls Born Healthy After Gene Surgery As Single-Cell EmbryosThe He Lab2018-11-26 | Two Chinese girls, who we'll call Lulu and Nana to protect their privacy, were born healthy a few weeks ago. Their mother Grace started her pregnancy by regular IVF with one difference: right after sending her husband's sperm into her eggs, an embryologist also sent in CRISPR/Cas9 protein and instructions to perform a gene surgery intended to protect the girls from future HIV infection. The surgery reproduces a natural genetic variation shared by more than 100 million people of primarily European origin that confers strong resistance to initial HIV-1 infection and disease progression. While CRISPR/Cas9 has been studied in human cells and in early clinical trials, gene surgery in embryos intended for pregnancy has not previously been reported. Safety remains a key concern, particularly for unintended changes to the genome. To assess the girls' genomes for safety after the surgery, multiple whole genome and targeted deep sequencing techniques were used before implantation, during the pregnancy, and now after birth. These data indicate the girls' genomes' were changed as intended by the gene surgery, but no off-target editing or large deletions occurred. Further assessments to confirm these findings will be conducted over the next year. We will publish our full data soon.
Email: HeJiankuiLab@gmail.com He Jiankui Lab Website: http://www.sustc-genome.org.cnDraft Ethical Principles of Therapeutic Assisted Reproductive TechnologiesThe He Lab2018-11-26 | Five Core Principles for Genome Editing in Human Embryos
1. Mercy for families in need (悲悯之心) A broken gene, infertility, or a preventable disease should not extinguish life or undermine a loving couple’s union. For a few families, early gene surgery may be the only viable way to heal a heritable disease and save a child from a lifetime of suffering.
2. Only for serious disease, never vanity (有所为更有所不为) Gene surgery is a serious medical procedure that should never be used for aesthetics, enhancement, or sex selection purposes — or in any way that would compromise a child’s welfare, joy, or free will. No one has a right to determine a child’s genetics except to prevent disease. Gene surgery exposes a child to potential safety risks that can be permanent. Performing gene surgery is only permissible when the risks of the procedure are outweighed by a serious medical need.
3. Respect a child’s autonomy (探索你自由) A life is more than our physical body and its DNA. After gene surgery, a child has equal rights to live life freely, to choose his or her occupation, to citizenship, and to privacy. No obligations exist to his or her parents or any organization, including paying for the procedure.
4. Genes do not define you (生活需要奋斗) Our DNA does not predetermine our purpose or what we could achieve. We flourish from our own hard work, nutrition, and support from society and our loved ones. Whatever our genes may be, we are equal in dignity and potential.
5. Everyone deserves freedom from genetic disease (促进普惠的健康权) Wealth should not determine health. Organizations developing genetic cures have a deep moral obligation to serve families of every background.Designer Baby is an EpithetThe He Lab2018-11-26 | A child is not something to be designed. That is not what these parents want to do. They carry lethal or debilitating diseases, often a small mistake in a single gene. It is inhuman for them not to protect their children if nature gives us the tools to do so. If you must call these babies something, call them gene surgery babies. Or better yet, a person who had to undergo an early gene surgery to protect their life.
You can read our principles for ethical gene surgery early in life via our publication in The CRISPR Journal: "Draft ethical principles for therapeutic assisted reproductive technologies."
We will publish our full data soon.Why We Chose HIV and CCR5 FirstThe He Lab2018-11-26 | For the first gene surgery in embryos, the He lab chose one specific gene surgery based on safety and real world patient benefit. First, the gene we chose, CCR5, is one of the best studied genes. More than 100 million people naturally have a genetic variation that disables CCR5, protecting them from HIV. Our surgery reproduces this variation to disable CCR5. Decades of clinical trials have targeted CCR5, including the first gene editing trial in the United States. There are also technical factors, such as how distant the gene is from other genes, that improve the safety profile of this gene for editing. Second, HIV remains a deadly disease and a top cause of death in many countries, although not in the United States and Europe. New infections remain more than three times higher than UNAIDS goals for 2020. Discrimination can make an infection a more serious threat to life.
We will publish our full data soon.
He Jiankui Lab Website: http://www.sustc-genome.org.cnGene Surgery in Embryos: An Embryologist Explains How It WorksThe He Lab2018-11-26 | Embryologist Qin Jinzhou at the He Jiankui lab explains how gene surgery in single cell embryos works. Dr. Qin conducted several years of preclinical studies on non-viable human embryos, donated by couples undergoing IVF, to assess the safety and feasibility of gene surgeries using CRISPR/Cas9 genome editing to prevent HIV and a severe high cholesterol disease that causes heart attack as early as age four.
We will publish our full data soon.
He Jiankui lab website: http://www.sustc-genome.org.cn.