CRISPR-Cas9: England takes giant leaps in genome-editing on human embryos

by NexDev | January 14, 2016 | Bioethics

On Friday September 18, 2015, Kathy Niakan, scientist working on stem cells at the Crick Francis Institute in London, had requested the “Human Fertilization and Embryology Authority » (HFEA), the U.K. governmental regulatory organization for fertility techniques, a license to perform genome-editing on human embryos.

On January 14, the British regulatory committee is assessing the request. In a press conference, Kathy Niakan went over the reasons for her request. The scientist conducts destructive research on human embryos derived from in vitro fertilizations and which are not wanted. She studies the fertilized ovule transfer to blastocyst, a structure which is then implanted in the mother’s uterus. The blastocyst contains several types of cells: those intended to become the fetus and those which become the placenta and the yolk sac. Kathy Niakan tries to understand fundamental biological aspects in the human embryos’ early development and the role of some specific genes, which could have clinical implications in infertility, miscarriages, development problems, and therapeutic applications of embryonic stem cells.

The researchers have already identified several thousand genes which are active in the embryo, the scientist and her team are working to identify those which are the principle regulators of development, and hope to employ the CRISPR-Cas9 technique to inactivate or activate the genes, and thus better understand their specific functions. The human embryos used for this purpose would be destroyed within seven days of development.

If the HFEA grants this request, Kathy Niakan hopes to start this research in a few months time, even if the project must also be approved by the British equivalent of an institutional examining committee.

During the press conference, Kathy Niakan was accompanied by her close collaborator Robin Lovell-Badge, very pro-active on this issue in the United Kingdom and in international debates on human gene editing. He has been on the organizational committees for the international summit in Washington and the joint statement in support of research and funding methods of gene editing by the Academy of medical science and several counsels and associations signed on September 2, 2015.

The director at Nuffield Council on Bioethics, the more reserved Hugh Whittall, declared: “The DNA editing performed for research purposes cannot be used for treatment purposes. However, this opens the doors to scenarios of gene editing to correct a genetic disease with the idea of creating a genetically modified embryo in order to give birth to a child. This research gives rise to a number of important questions which need to be addressed before research work begins.”

The Nuffield Council on Bioethics is currently achieving an online public survey to clarify the examination of ethical questions raised by this technique of gene editing.

England is thus advancing by giant leaps and bounds on the question of employing CRISPR-Cas9 for gene-editing on human embryos. For Alliance VITA, this is a major preoccupation. Using the human embryo simply as ‘raw material’ for research raises serious ethical questions. Although this technique might be promising for genetic therapy, Tugdual Derville reminds us that when applied to the human embryo: “the danger is to cause the emergence of custom-made babies, with pre-selected genetic criteria, transmissible modifications, with unknown consequences for future generations. The human genome is part of our most precious “heritage of humanity”. Its’ integrity must absolutely be preserved for future generations.”

Alliance VITA kicks off its 11th Bioethics Conference

by NexDev | January 12, 2016 | Bioethics

This past Monday, January 11, Alliance VITA kicked off its 11th Bioethics Conference on the theme “Our society in need of healing – How to act in favor of a culture of life?”

Thanks to the network of VITA volunteers engaged all over France and even abroad, there are nearly 112 cities throughout France and a dozen cities abroad which organize this event this year on local levels. More than 5,000 participants were able to follow the invited speakers’ conferences by videoconferencing, which were broadcast live from the packed Parisian hall.

The first evening was dedicated to the « force of personal action ». Encouraged by the experts’ presentations and national witnesses, each participant was invited to perform a distinctive reflection on his personal background, analyzing the events that marked his life which could be an incentive for a personal commitment.

During the general introduction for the training cycle, Alliance VITA’s president, François-Xavier Peres gave witness to his personal “bioethical background”. Then, the philosopher Martin Steffens, author of the “Little tract of joy: consent to life” (2013 Christian Humanism Prize) and of Life in Blue (2014), shared his philosophical reflections and his own experience on hardships and difficulties in life. He encouraged the participants to reflect on personal strengths that each could deploy when confronted with hardship.

The consultant, Anne Davigo Le Brun, specialized in personal accompaniment, then shared her expertise on bereavement by addressing some crucial questions: what stages does grieving lead us through? How can we support a grieving individual? What does bereavement teach us about ourselves, about our humanity? Can life forces emerge from bereavement?

The evening continued with the interview of the first « important founder” (there will be one each evening): Laurent de Cherisey, who created the Simon of Cyrene community – which are living areas shared between individuals with cerebral brain-damage and non-disabled people. Latest prizewinner of “France is committed”, a new title created by the presidential initiative to support innovative projects, Simon of Cyrene is above all a place which puts “the encounter” at the heart of its’ project for life. Laurent de Cherisey taught us that “the disabled person helps us take off our masks, to advance, not by fear of the other, but by fear of oneself. Very often, a disabled person’s greatest suffering is not his disability, but his loneliness.”

Tugdual Derville, Alliance VITA’s general deputy, finished the speeches by presenting the “figure of Moses, as a transmitter of life”. The history of the survival of this baby reminds us that protecting life is a right sealed in the inviolable conscience of every human being. The fact that he is successively saved by 5 women leads us to reflect on the maternal instinct of each one of them, to the anthropology of the gift that it reveals, and to see every woman as a carrier of life, of history and of transmission. The life and the mission of this figure which is part of the heritage of humanity reveals the importance of the “inner self”, the necessity to follow one’s conscience and to respond to the missions which are entrusted to us.

The participants also discovered and exchanged viewpoints on a poignant extract from the film “Human”. Alternating with times for reflection and activities, the evening was concluded with a round-table discussion where each of the 5 invited speakers in turn, could respond to questions from the interconnected cities.

>> Note: Join us Monday January 18, for the 2^nd evening dedicated to “Humanitarian action towards the most

CRISPR-Cas9 and new promising advances for Duchenne muscular dystrophy

by NexDev | January 8, 2016 | Bioethics

The revolutionary genetic technique CRISPR-Cas9 is gaining more and more attention. Some new scientific studies have just been published which present the work which has been carried out in treating a very serious pathology, thus far, unfortunately considered incurable: Duchenne myopathy.

This neuromuscular disease is marked by severe muscular weakness, and progressive pathological atrophy in all body muscles. This genetic disease is set off by gene mutations of the DMD gene, which codes for an essential muscular functioning protein: dystrophin. This very large gene is carried by chromosome X.

Three independent research teams working on this subject, published simultaneously in the review Science the result of their work carried out on mice with CRISPR-Cas9, a technique which opens unprecedented possibilities for correcting those mutations. This genetic engineering technique allows targeting DNA zones to be cut out in order to inhibit or replace the defective gene. Thus the three teams worked on cutting out the defective gene in the DNA sequence concerned. In the absence of the defective DNA segment, the muscular cell produces a shorter, yet functional dystrophin protein, visibly allowing the mice to recuperate some strength.

At first, one of the teams performed this genome modification experiment on a mouse embryo (fertilized egg), thus creating a subject considered to be healthy. But this modification becomes transmissible to future generations, and these experiments do not respond to the needs of patients with muscular dystrophy. This team then worked on modifying the genome of the muscular cells of the young mouse. To do this, they used an inoffensive virus carrying the genome modification system capable of infecting the muscular cells in the entire body of the mouse to depose in each one these “scissors” used to cut out the DNA. The cells of the genome thus “repaired” then started coding a functional dystrophin.

The two other teams conducted similar experiments, targeting other zones of the defective gene. But one of the teams, decided to go even further, by using a virus capable of carrying the CRISPR-cas9 equipment to the heart of the germ cells which produce the new muscular cells, to modify the genome. Treating the muscular germ cells of a patient could achieve a more permanent therapeutic result, than the correction of ordinary, mature muscular cells, which are renewed at a rapid pace in patients with muscular dystrophy compared to patients who do not suffer from this disease.

The three teams filed patents. Even if a genetic therapy for muscular dystrophy can be envisioned, there is still considerable work to be done before human clinical trials can begin. It remains to be seen: how the human immune system will react to components from the gene modification system or to the modified dystrophin proteins to which it is not accustomed.

Les Echos, a daily newspaper, underlines that “At the forefront of the fight against genetic diseases, genome modification constitutes a formidable weapon in the hands of doctors.” This genome modification technique is revolutionary and lets one imagine countless potential therapies, as in these studies. It depends on how the technique is used.

Let us keep in mind that the CRISPR-Cas9 technique leads to major ethical questions if used on human embryos or human germ cells. “It’s a red line not to be crossed” recently recalled, Tugdaul Derville, Alliance VITA’s General Deputy. “Applying these techniques of editing DNA to human reproductive cells or human embryos legitimately causes dizziness. We are risking the emergence of custom-made babies and the modification, in a transmissible manner, of the human genome, which constitutes a world heritage of humanity and which therefore deserves all our necessary precautions.”