China claims to have created the World’s first designer twins

China’s claim of using gene-editing to create the World’s first designer babies have stirred outrage in the scientific world. The researchers fear that it will stall their efforts to translate gene-editing technology into safe treatments for genetic diseases.

He Jiankui is a Stanford and Rice University-trained physicist and claims to have used CRISPR-Cas9 to delete the embryos’ CCR5 gene (C-C motif chemokine receptor 5), which is linked to resistance to HIV infection and the twins were born a few weeks ago with innate immunity to HIV, the virus that causes AIDS.

He Jiankui

In 2010, when Jiankui was a Rice University student, he already published a paper on details about gene-editing using the bacterial immune system CRISPR. It was well before Geneticist Jennifer Doudna co-invented the groundbreaking current technology for editing genes, called CRISPR-Cas9.

He currently works at the Southern University of Science and Technology of China in Shenzhen and has posted videos to YouTube about his editing techniques. The research is not yet published in a peer-reviewed journal and is, however, yet to be verified by outside scientists and researchers.

The news stunned the scientist and researchers all over the globe as they prepare to gather in Hongkong for the Second International Summit on Human Genome Editing from November 27-29, 2018. David Baltimore, Nobel laureate and chair of the organizing committee said, “I don’t know the details.”

 “We don’t know what will be said,” when He speaks at a session on human embryo editing on Tuesday, November 27, 2018.

There are two main issues with the existing gene-editing technique if used to create ‘designer babies’ in the real world. The first is mosaicism, in which the edited gene does not make it way into every cell of the embryo and second are off-target effects, where other parts of the genome may get deleted accidentally, and the consequences remain unknown.

Before genome editing is used as medical treatment, it is essential that the issues of mosaicism and off-target effects are resolved. Long-term follow-up of babies who have had gene-editing done is needed to know about long-term effects.

It is also unclear that why this particular gene was editing in the twins, but reports coming out of China suggest that the father was HIV positive, but his infection was under control and mother was HIV negative. The scientists also fail to understand that why he chose the HIV gene for editing, as there already exist proven treatments to prevent HIV-positive parents from infecting their children.

There are also specific risks of having a disabled CCR5 gene, which includes a higher risk of West Nile virus infection and dying from Influenza.

The summit’s organizing committee issued a statement Monday saying they had only just learned of He’s research in Shenzhen, China. “Whether the clinical protocols that resulted in the births in China conformed with the guidance” of leading scientific bodies for conducting clinical trials of heritable genome editing “remains to be determined,” the statement said. “We hope that the dialogue at our summit further advances the world’s understanding of the issues surrounding human genome editing. Our goal is to help ensure that human genome editing research is pursued responsibly, for the benefit of all society.”

Meanwhile, Chinese officials have condemned He’s research and strongly oppose the use of gene-editing technology in humans. gene-edited babies are prohibited in China. The Ethical Guiding Principles for the Research of Human Embryonic Stem Cell which was published by the Chinese government in 2003 states that scientists are allowed to conduct genetic editing to human embryos only for research purposes, but the time for their in-vitro breeding shall not exceed 14 days from the date of fertilization or nucleus transplant.

Here is a YouTube video showing He giving information about the twins

Other YouTube videos can be watched here

Statement from the Organizing Committee on Reported Human Embryo Genome Editing

Ob/Gyn Updated Facebook page

How crisper works? Excellent 2 minutes video about CRISPR technology

courtesy: ISTOCK

CRISPR or clustered, regularly interspaced, short palindromic repeat (CRISPR) is a versatile tool for genome engineering used for generating RNA-guided nucleases, such as Cas9, with customizable specificities.

The tool allows scientists to make precise edits to DNA strands, which could lead to treatments for genetic diseases ... but could also be used to create so-called "designer babies."

It has already been used for human trait selection in the UK, as we have seen in the three-parent in vitro fertilization for eliminating mitochondrial diseases.

Here is a beautiful, short two minutes video by STAT, explaining how CRISPR works. 

US National Academics report paves the path forward for genetically modified babies with proper guidelines.

Courtesy: Business insider 

  

The US National Academies of Science, Engineering, and Medicine today submitted a report laying down guidelines for  genetically modifying human embryos to eliminate devastating genetic conditions.

This 261page report follows the conclusion of 2015 International Summit on Human Gene Editing. It took multidisciplinary committee experts a full year to perform in depth review of literature, monitoring the latest advancement and getting input from all the prominent players in field of human gene editing.

The committee set forth specific criteria before experimentation and clinical trials can begin regarding these heritable germline  mutation. The criteria for heritable germline editing by National Academy of Science and National Academy of medicine are:[1]

1. Absence of reasonable alternatives
2.  Restriction to preventing a serious disease or condition
3.  Restriction to editing genes that have been convincingly demonstrated to cause or to strongly predispose to the disease or condition
4. Restriction to converting such genes to versions that are prevalent in the population and are known to be associated with ordinary health with little or no evidence of adverse effects
5. Availability of credible pre-clinical and/or clinical data on risks and potential health benefits of the procedures
6. Ongoing, rigorous oversight during clinical trials of the effects of the procedure on the health and safety of the research participants
7.  Comprehensive plans for long-term, multigenerational follow-up while still respecting personal autonomy
8. Maximum transparency consistent with patient privacy
9. Continued reassessment of both health and societal benefits and risks, with broad on-going participation and input by the public
10. Reliable oversight mechanisms to prevent extension to uses other than preventing a serious disease or condition

At present genome editing, should not be used for creating designer human beings  with all the chosen, positive  traits.

Alta Charo, a bioethicist at the University of Wisconsin–Madison and co-chair of the report said “Up until now, we’ve been talking only hypothetically and most people assumed we simply wouldn’t ever do this. We are not saying that you have to or you should, but we are saying that if you can meet these criteria it is permissible.”

Many researchers who opposed the gene editing technique where disappointed by the green signal.

George Church, a geneticist at Harvard University in Cambridge, Massachusetts opined that it is difficult to draw a line between medical use and creating ‘super humans’. 

Scientist also expressed concerns over ‘Medical tourism’ as scientist working in countries with less stringent regulation will start modifying embryos for purpose other than medical reasons. 

The complete report can be download here

The summary of recommendations can be found here

The principles for the governance of Human Genome Editing can be found here.  

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[1] http://nationalacademies.org/cs/groups/genesite/documents/webpage/gene_177255.pdf

CRISPR, a new genome editing tool- Ted talk by Geneticist Jennifer Doudna

CRISPR, a new genome editing tool, has the potential to cure genetic disease, potentially opening the road to "engineered humans."

Geneticist Jennifer Doudna co-invented a groundbreaking new technology for editing genes, called CRISPR-Cas9. The tool allows scientists to make precise edits to DNA strands, which could lead to treatments for genetic diseases ... but could also be used to create so-called "designer babies." Doudna reviews how CRISPR-Cas9 works — and asks the scientific community to pause and discuss the ethics of this new tool.

It has already been used for human trait selection, as we have seen in the three-parent in vitro fertilization for eliminating mitochondrial diseases.

Here is a   Ted talk by her regarding what is  CRISPER-Cas9 technology, the ethical and societal implication of the procedure in future.

Designer babies: an ethical horror or scientific boon

Designer babies: intelligent, perfect on all account with no flaws is no longer a part of science fiction. They are real and here to stay. The latest development of crisper technology has made it possible to design embryos according to the demand.

As scientific and ethical arguments over ‘three parent babies’  go on, designer babies are not far behind in the line.

Creating genetically modified people is no longer a science fiction fantasy; it's a likely future scenario. Biologist Paul Knoepfler estimates that within fifteen years, scientists could use the gene editing technology CRISPR to make certain "upgrades" to human embryos — from altering physical appearances to eliminating the risk of auto-immune diseases. In this thought-provoking talk, Knoepfler readies us for the coming designer baby revolution and its very personal, and unforeseeable, consequences.

A worth listening Ted talk by the famous biologist Paul Knoepfler.