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Nobel Prize for Medicine 2023

Portraits dessinés des deux lauréats du prix Nobel de médecine 2023 : Katalin Karikó (àgauche) et Drew Weissman (à droite)Ill. Niklas Elmehed © Nobel Prize Outreach 

The winners of the 2023 Nobel Prize in Physiology or Medicine were announced on Monday October 2, 2023. They are American-Hungarian Katalin Karikó and American Drew Weissman. They are being rewarded for their discoveries concerning nucleic base modifications, which were essential to the development of effective messenger RNA vaccines against Covid-19. Their work has also fundamentally altered our understanding of how mRNA interacts with our immune system.

 

At Inserm, research into the many potential applications of messenger RNA continues.

 

Find out more about this topic online:

Nobel Prize 2020

Only available in french

Malaria: Vaccine clinical trial for Pregnant Women yields promising results

©Benoît Gamain. Gestational malaria is associated with low birth weight for the baby and an over-risk of neonatal mortality.

Malaria infection during pregnancy represents a major public health problem in the regions endemic for the disease, substantially increasing the risks to mothers and their unborn children. For newborns, malaria is linked to low birth weight and an excess risk of mortality. To protect this population, a team of researchers from Inserm and Université de Paris led by CNRS Research Director Benoît Gamain is developing a vaccine at the French National Institute of Blood Transfusion (INTS). Called PRIMVAC, the vaccine has undergone a clinical trial to study its safety and collect preliminary data on its ability to induce an immune response. The results of this clinical trial sponsored by Inserm[1] have been published in the prestigious journal Lancet Infectious Diseases.

According to the World Health Organization, malaria is responsible for over 400,000 deaths each year. Despite the progress made in fighting the disease in recent decades, some populations remain particularly vulnerable. One such population is pregnant women.

In the areas of the world where malaria is endemic, people acquire immunity throughout their childhood, meaning that they are generally protected against its most severe outcomes once they reach adulthood. However, pregnant women are an exception because the red blood cells infected with the Plasmodium falciparum parasite responsible for malaria accumulate in the placenta, promoting anemia and gestational hypertension. The disease is also linked to a higher risk of spontaneous abortion, premature birth and intrauterine growth delays which lead to low birth weight and a high rate of neonatal mortality. In Sub-Saharan Africa, 11 million pregnant women were infected with malaria in 2018, with around 900,000 of their babies born underweight.

To tackle this public health problem, a team of researchers from Inserm and Université de Paris led by CNRS Research Director Benoît Gamain has spent the past two decades developing a vaccine for gestational malaria. The goal is to prevent the deaths of up to 10,000 mothers and 200,000 babies each year. “Developing an effective vaccine for young women before their first pregnancy is a priority if we are to reduce malaria-related mortality. An effective strategy could focus on a population similar to that targeted by HPV vaccination, for example, before the women become sexually active”, emphasizes Benoît Gamain.

A safe and effective vaccine

Called PRIMVAC, the vaccine had recently been produced in large quantities in accordance with current regulations. In a clinical trial published in Lancet Infectious Diseases, the researchers provide data on its safety and ability to induce an appropriate immune response, up to 15 months after the initial vaccination.

The vaccine was evaluated in 68 non-pregnant women aged 18 to 35 at the Cochin Pasteur Clinical Investigation Center in Paris, then at the National Center for Research and Training on Malaria (CNRFP) in Ouagadougou, Burkina Faso. The participants were randomly assigned to 4 cohorts, receiving the vaccine at various doses, on 3 occasions over a period of 3 months. These women were then monitored for 15 months in order to identify and treat any side effects and study the immune response induced by the vaccination.

Antibody (green) of a vaccinated volunteer binding to the surface of a human red blood cell infected with the Plasmodium falciparum parasite (blue). Credits: Inserm/Chêne, Arnaud et Semblat, Jean-Philippe

The results of this study show that PRIMVAC is well tolerated. In addition, the researchers have shown that vaccine can produce an immune response, with the production of antibodies in 100% of women vaccinated after only two injections. The antibodies produced are capable of both recognizing the parasitic antigen on the surface of the infected red blood cells and inhibiting their adhesive capacity, which is responsible for their accumulation in the placenta.

“We were able to show that the vaccine is well tolerated, at all the tested doses. The side effects observed were mainly pain at the injection site. We also revealed that the quantity of antibodies generated by the vaccine increases after each vaccination and that they persist for several months. It therefore appears that the vaccine has the capacity to trigger a lasting and potentially protective immune response”, underlines Gamain.

Studying this immune response on the longer term will be the subject of future clinical trials. The researchers want to continue monitoring the 50 Burkinabe volunteers in order to evaluate whether the immune response induced by the vaccination is maintained until their first pregnancy.

 

[1] The trial was coordinated by the Cochin Pasteur Clinical Investigation Center in Paris and the EUCLID/F-CRIN clinical trials platform in Bordeaux in collaboration with the National Center for Research and Training on Malaria (CNRFP) in Ouagadougou, Burkina Faso, and the European Vaccine Initiative (EVI). Funding: Federal Ministry of Education and Research, through the development bank KfW, Germany; Inserm, and National Institute of Blood Transfusion (INTS), France; Irish Aid, Department of Foreign Affairs and Trade, Ireland.

“Fake news” and misinformation around the SARS-CoV2 coronavirus

[credits]Adobe Stock

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As with all highly mediatized topics, SARS-CoV-2 has its share of misinformation. Originating in the Chinese province of Hubei in 2019, the epidemic caused by this novel coronavirus, now referred to as SARS-CoV-2, and COVID-19 for the disease it causes in people, continues to stoke the fires of not just the traditional media, but also websites and social media – and not always to the good. We at Inserm suggest taking a look at the various forms that this wave of misinformation can take, so that you can focus on what is relevant.

From conspiracy theories and unfounded revelations to the plain and simple approximation of figures, when it comes to SARS-CoV-2 and its human disease COVID-19, there is no shortage of misinformation and fake news communicated through numerous media channels on subjects ranging from the nature of the virus to its vaccine.

More information on  SARS-CoV-2 can be found here (in French): https://www.inserm.fr/information-en-sante/dossiers-information/coronavirus-sars-cov-et-mers-cov

The virus itself

There are many questions as to the nature of this coronavirus and its public health implications. Is it safe to receive packages coming from or via China? Does the virus affect only elderly people? Do some foods help prevent infection? Is the incubation period longer than initially thought?

Although some gray areas still surround SARS-CoV-2, we know that the virus is unable to survive on objects, such as letters and cardboard boxes, sent long distance for the time it takes for them to be reach their recipients. We also know that transmission of the virus is essentially airborne, via droplets or aerosols of saliva projected by coughing or sneezing. Therefore, an asymptomatic person (who is contaminated but is not coughing or sneezing close to others) has a lower risk of spreading the virus than a symptomatic person, except where there is close and/or prolonged physical contact. Also, the virus can be transmitted through contact with contaminated surfaces if the person touching the surface then touches their face without washing their hands first. Therefore, frequent hand washing is very important. What is more, one’s geographic or ethnic origin has no bearing on the ability of the virus to infect others.

Contrary to what might have been said in recent weeks, there are no foods (garlic, fennel) or hygiene products (sprays, mouthwashes) that prevent infection with SARS-CoV-2 . Finally, the average estimated incubation period is from 5 to 6 days, with a period of 1 to 12 days for the majority of cases. Research is continuing in order to clarify both the unanswered questions and unfounded assertions.

A vaccine for the Covid-19 epidemic?

A vaccine to prevent the contraction of SARS-CoV-2 does not exist yet. The various existing vaccines used to prevent bacterial pneumonia are unfortunately ineffective. Teams across the world, including an Inserm team in Lyon, have rallied in attempts to understand this novel virus and counter it as quickly as possible, although that will take some time. Contrary to what can be read on the more or less conspiracy theorist websites, which insinuate that a vaccine is already ready and that pharmaceutical companies are speculating on the deadliness of the epidemic in order to push up prices, the development of a vaccine is still under study – this is why the only means of protection offered to healthy populations are barrier measures.

Conspiracy theories surrounding a coronavirus patent

The conspiracy theories keep coming. Some are claiming that SARS-CoV-2 was created in the P4 laboratory in Wuhan, or that pharmaceutical companies had already filed patents to cash in on the sale of a specially-developed vaccine that will be withheld until millions have died in order to push prices up. However, the patents in question, found online, are patents for the Chinese SARS coronavirus (SARS-CoV) of 2002, which was logically then studied in order to enable the development of a vaccine. These are totally untrue.

In particular, a conspiracy theorist video pointing the finger at Inserm and Institut Pasteur is currently doing the rounds on social media. The interpretation of the documents presented in this video is totally erroneous and the allegation that Institut Pasteur had invented the virus responsible for COVID-19 is false. The 2004 patent held up as “evidence” in this video does not describe the invention of SARS-CoV-2 responsible for COVID-19, but rather the discovery of SARS-CoV-1 responsible for SARS, and the invention of a vaccine strategy following the epidemic of the latter that originated in China in 2002. This candidate vaccine against SARS-CoV-1 was not tested in humans because the epidemic ended before clinical trials could be implemented, with the lack of patients rendering them impracticable. The knowledge acquired from fighting this epidemic of a virus related to the present-day coronavirus is currently being actively used by the researchers concerned in order to create a potential vaccine against Sars-CoV-2. More details can be found on the Institut Pasteur website: https://www.pasteur.fr/en/coronavirus-institut-pasteur-warns-against-false-information-circulating-social-media

“Fake news” and other myths busted by the World Health Organization:https://www.who.int/emergencies/diseases/novel-coronavirus-2019/advice-for-public/myth-busters

Here are a few definitions of the expressions used by researchers and communicated by the media:

What is a preprint? A preprint is a paper written by researchers that has not yet undergone the standard process for publication in a scientific journal (revision, correction and peer review), which can sometimes be very lengthy. Preprints allow scientists to share research with each other in real time – in this case concerning the SARS-CoV-2 epidemic – and can be found on the Biorχiv and Medrχiv websites (which stand for Bio Archive et Med Archive, respectively).

In scientific documents such as patents, what does the term “inventor” mean?

In research, someone who discovers a virus, compound or geological deposit, for example, is designated as its “inventor”. The discovery is designated as the “invention”. In the case of the aforementioned 2004 patent, the designated “inventors” of SARS-CoV-1 that was responsible for SARS are the people who discovered and described it – they did not create it.

How are cases of COVID-19 defined? 

The definition of suspected cases of COVID-19 changes with time and with the spread of the epidemic. The criteria also vary from one country to another. In France, the criteria established by its public health agency, Santé publique France, are regularly updated.

See the SARS-CoV-2 information page provided by Santé publique France  (only available in French).

Do I need to get a prescription for an antimalarial to fight coronavirus?

For the moment there has been no firm scientific validation as to the efficacy of chloroquine antimalarial treatment against coronavirus. The research mentioned in the press concerns a very small study whose methodological validity is controversial.

Although the possibility of being able to use safe, well-known and inexpensive antimalarial treatments in coronavirus patients is an interesting one, it is essential to have visibility over the data produced by clinical trials involving SARS-CoV-2 patients, published and therefore made accessible to the international scientific community.

A European clinical trial evaluating four experimental treatments for COVID-19 began on March 22, 2020. Coordinated by Inserm as part of the REACTing consortium, this trial will enroll at least 800 French patients with severe forms of COVID-19: https://presse.inserm.fr/en/lancement-dun-essai-clinique-europeen-contre-le-covid-19/38737/

It must also be noted that taking any kind of medication is not without risk. Antimalarials can only be obtained on medical prescription. 

Deadly outbreak in China linked to a novel form of coronavirus

 

Map of Chinese provinces colour coded according to the number of cases of 2019-nCoV [4] as at 27 January 2020. Credits Vittoria Colizza

 
Inserm is a signatory to the following international joint statement issued by the Wellcome Trust [1] :

Sharing research data and findings relevant to the novel coronavirus (nCoV) outbreak
 
The outbreak of the novel coronavirus in China (2019-nCoV) represents a significant and urgent threat to global health.
 
We call on researchers, journals and funders to ensure that research findings and data relevant to this outbreak are shared rapidly and openly to inform the public health response and help save lives.
 
We affirm the commitment to the principles set out in the 2016 Statement on data sharing in public health emergencies, and will seek to ensure that the World Health Organization has rapid access to emerging findings that could aid the global response.
 
Specifically, we commit to work together to help ensure:
 
all peer-reviewed research publications relevant to the outbreak are made immediately open access, or freely available at least for the duration of the outbreak
research findings relevant to the outbreak are shared immediately with the WHO upon journal submission, by the journal and with author knowledge
–  research findings are made available via preprint servers before journal publication, or via platforms that make papers openly accessible before peer review, with clear statements regarding the availability of underlying data
–  researchers share interim and final research data relating to the outbreak, together with protocols and standards used to collect the data, as rapidly and widely as possible – including with public health and research communities and the WHO
–  authors are clear that data or preprints shared ahead of submission will not pre-empt its publication in these journals
 
We intend to apply the principles of this statement to similar outbreaks in the future where there is a significant public health benefit to ensuring data is shared widely and rapidly.
 

Coronaviruses, which are named after the crown shape of the proteins that surround them, are a family of viruses that have a traditionally animal reservoir.

In some cases, transmission from animal to human is possible, as the SARS, MERS and probably this new virus have shown.

While these viruses are mostly benign in humans, two death-causing epidemics have been reported in recent years in the Middle East (MERS-Cov, 2012-2013) and in China ten years earlier (SARS-Cov, 2002-2003).

For this third outbreak, the outbreak would come from a fish and seafood market in Wuhan, where live animals are also sold. The search for the reservoir and host is ongoing.

Inserm is already mobilized to respond to the epidemic, under the action of the Reacting consortium, which brings together French research groups of excellence. Several initiatives have been launched:

  • The consolidation of a «modelling group» set up at the request of Reacting, to model the possible spread of the epidemic in France and Europe, and anticipate its impact on populations.

 

  • The work entrusted to Bruno Lina (Inserm Unit 1111 – Centre International de Recherche en Infectiologie, CIRI) to propose a research project on potential therapeutic targets to identify molecules capable of acting on several types of coronavirus.

 

  • Support for clinical research, through the dissemination to Reacting’s French and international partners of best practices in research protocols/ standardization of data collection.

[1] List of signatories : Academy of Medical Sciences, UK; African Academy of Sciences; Assistant Secretary for Preparedness and Response / Biodefense Advanced Research and Development Authority, USA; Austrian Science Fund (FWF), Bernhard Nocht Institute for Tropical Medicine (BNITM); Bill & Melinda Gates Foundation; BioRxiv; Biotechnology and Biological Sciences Research Council (BBSRC), UK; Bulletin of the World Health Organization; The British Medical Journal (BMJ); Calouste Gulbenkian Foundation – Gulbenkian Science Institute; Canadian Institutes of Health Research; Centers for Disease Prevention and Control (CDC), USA; Chinese Centre for Disease Control and Prevention; The Department for International Development (DFID); DG Research & Innovation, European Commission; Dutch Research Council (NWO); eLife; EcoHealth Alliance; EMBO; Epicentre – MSF; European Respiratory Journal ; ERJ Open Research; F1000 Research Limited; Fondation Merieux; Food & Drug Administration, USA; French National Research Agency (ANR); The Global Health Network; Global Virus Network; GLOPID-R ; Inserm (Institut national de la santé et de la recherche médicale), France ; The Institut Pasteur; International Severe Acute Respiratory and emerging Infection Consortium (ISARIC); International Society for Infectious Diseases (ISID); Japan Agency for Medical Research and Development (AMED); Johnson & Johnson; The Lancet Luxembourg National Research Fund (FNR); Medical Research Council (UK); Médecins Sans Frontières/Doctors Without Borders (MSF); MedRxiv; Merck Research Laboratories; Microbide Limited National Academy of Medicine, USA; National Institutes of Health (NIH), USA; National Institute for Infectious Diseases Lazzaro Spallanzani, Italy; New England Journal of Medicine; Office of Global Affairs, Department of Health and Human Services, USA; Portland Press; PLOS; The Research Council of Norway; The Royal Society; Science Europe; Science Foundation Ireland ; ScienceOpen; South African Medical Research Council Takeda; Taylor & Francis; ZonMW – The Netherlands Organisation for Health Research and Development

Inserm is wishing you a Happy Holiday!

L'Inserm vous présente ses meilleurs voeux pour l'année 2020

Reprise de la campagne de vaccination contre la grippe

 

D’après la Fédération des pharmaciens d’officine (FSPF), plus d’un million de personnes en France s’étaient déjà faites vaccinées contre la grippe au 1er novembre 2019, deux semaines après le lancement de la campagne annuelle. Avec le réseau Sentinelles et le projet Grippenet, l’Inserm participe activement à la surveillance des apparitions de symptômes grippaux sur le territoire français.

Entamée le 15 octobre, la campagne de vaccination contre la grippe 2019 continue de s’étendre, les pharmaciens dénombrant déjà plus d’un million de doses administrées au 1er novembre sur les 4 millions de doses délivrées. Dans notre dossier d’information sur la grippe, nous rappelons que c’est 2 à 8 millions de personnes qui sont touchées chaque année par le virus de la grippe, également appelé Influenzavirus.

Fatigue, fièvre, toux, « nez qui coule » : si les symptômes sont en apparence similaires à ceux d’un rhume ou d’une rhinopharyngite, la grippe est plus éprouvante que les autres pathologies fréquentes de l’hiver. Mutant chaque année, l’Influenzavirus peut même s’avérer mortel, notamment pour les enfants, les personnes âgées et les personnes fragiles, faisant en moyenne 5000 morts chaque année.

 

Sous haute surveillance à l’échelle mondiale, l’épidémie annuelle de grippe l’est aussi en France, sous la coordination de Santé publique France. C’est dans cet objectif que, depuis 1984, l’Inserm participe à la tenue du réseau Sentinelles, en partenariat avec la faculté de médecine de la Sorbonne. Plus de 1400 médecins généralistes et pédiatres libéraux, tous volontaires et répartis sur le territoire métropolitain, fournissent les données de leurs consultations afin d’établir un bilan statistique et géographique des tendances épidémiologiques. Les indicateurs ainsi fournis permettent d’évaluer l’incidence de nombreuses pathologies de manière hebdomadaire. On sait ainsi que les symptômes grippaux sont encore légers en cette fin de mois de novembre, même si certaines tendances se démarquent dans les Cévennes, dans l’Artois et dans le bocage mayennais.

Par ailleurs, les chercheurs et les professionnels de Santé peuvent aussi se reposer sur les données de surveillance fournis par le projet Grippenet. Mis en place en 2012 par le réseau Sentinelles, c’est un projet de surveillance épidémiologique permettant à tout le monde de participer en ligne, anonymement, en renseignant les symptômes dont ils pourraient souffrir d’une semaine sur l’autre. Cela permet aux chercheurs de l’Inserm et de Sorbonne Université de suivre l’évolution d’individus pouvant monter des symptômes grippaux sans pour autant avoir été consulter un médecin généraliste.

Bruno Lina, chercheur Inserm au Centre international de recherche en infectiologie (CIRI) de Lyon déclarait pour notre dossier que « [les] outils de lutte actuels ne suffisent pas à éliminer le problème de santé lié à la grippe. Nous avons un vrai besoin de connaissances dans tous les domaines ». Le réseau Sentinelles, avec l’appui du projet Grippenet, sont des outils importants pour les chercheurs désireux d’enrichir les connaissances épidémiologiques sur l’Influenzavirus et, entre autres, d’améliorer les mesures de prévention et l’efficacité des vaccins contre la grippe.

sentiweb.fr

Nobel prize 2019

Only available in french

Update : Jeanne Calment the oldest woman

Update on 16/09/2019

A new article published in the journal The Journal of Gerontology by Jean-Marie Robine, research director at Inserm and his Swiss and Danish colleagues, reaffirms that Jeanne Calment’s identity has not been usurped and that she died at the age 122 years old. In order to show this, they have:

– provided several mathematical models that conclude that there is a significant probability that Jeanne Calment reached the age of 122. “Thus, every 10 million centenarians, a person can reach 123 years of age.”writter the authors. A probability that is certainly small, but that is far from making Ms. Calment a statistical impossibility.

– identified the original elements that were used for the initial validation of Jeanne Calment’s identity and collected additional documentation. Cross-checking these numerous archives of various natures has shown that there was neither tax fraud nor falsification of Jeanne Calment’s identity.

In addition, Inserm collected the documents resulting from the investigation on supercentenarians conducted by Jean-Marie Robine and Michel Allard during the 1990s. These archives include written and audio documentation from Jeanne Calment, which is currently being archived and digitized at Inserm.

 

In the wake of the fraud allegations surrounding the age of Jeanne Calment which have received widespread mass media and social media coverage, Inserm as a leading life sciences and health research organization wishes to make clear the following:

– In 1998, following two years of research, Doctor Michel Allard from Fondation Ipsen and Jean-Marie Robine, demographer and public health researcher at Inserm published a letter in the journal Science, entitled The Oldest Human. Their findings, based on numerous civil and religious documents, supported the validity of Jeanne Calment’s death aged 122. Between them, the researchers have published several hundreds of articles in peer-reviewed scientific journals.

– The contribution of the Inserm researchers involved studying the quality of the documents available in the Arles archives (personal records, parish registries, census lists and school/military documents, etc.) and analyzing them with particular focus on the contribution of genetic factors to Jeanne Calment’s longevity. In doing so they revealed an extraordinary concentration of long-lived ancestors of Jeanne Calment and her brother François (Science 1998).

– The Fondation Ipsen study, A la recherche du secret des centenaires, for which Jean-Marie Robine had developed a specific protocol with Michel Allard, was the first to require age verification for all centenarian studies. This pioneering survey of the health and quality of life of the very elderly put an end to the use of convenience samples, ushering in the most modern epidemiological methods and questioning in the study of very old age. Since then, surveys concerning centenarians and nonagenarians endeavor to be as representative as possible. The Danish, Italian and Japanese surveys can be cited in this respect. The ongoing Five Country Oldest-Old People (5_COOP) study, also coordinated by Inserm, is based on a standardized survey conducted in the same way in Denmark, France, Japan, Sweden and Switzerland. It shows that not all centenarians are frail, suffering from dementia, or dependent and it illustrates the immense variation in health states at that age (Herr et al. 2018).

– Inserm proposes joining forces with the town of Arles in the immediate future to scan and make the genetic hypotheses and documents used to validate the lifespan of Jeanne Calment accessible to the community (Open Data).

– The researchers have 15 hours of recordings of Jeanne Calment, in the form of some thirty interviews conducted between 1992 and 1995. Before allowing public access, it must be ensured that they do not or no longer contain any information of a private or medical nature.

– Continuing the Open Science approach, an international database on supercentenarians (over 110) whose ages have been validated in 15 countries (including France) went online in 2010, marking the publication of the book Supercentenarians (www.supercentenarians.org). This database will in the near future be updated and extended to include those between the ages of 105 and 109 with the publication of a second book in the same collection. The data for the French section of this database is supplied by researchers from Inserm and INED.

– It is not within the remit of Inserm to support any requests for exhumation which do not fall within its field of competence.

– In scientific research, the burden of proof lies with those who propose new hypotheses or claim to have uncovered new findings. It is not for Inserm researchers to underpin the theory of the Russian objectors. Their arguments, should they have any, must be submitted to a peer-reviewed scientific journal which is responsible for validating the robustness of the research in question.

References

Robine J.M., Allard M. The oldest human. Science. 1998. Mar 20;279(5358):1831.

Robine J.M., Allard M. Jeanne Calment: validation of the duration of her life. In: Jeune B, Vaupel JW, editors. Validation of exceptional longevity. Odense monographs on population Aging; 6. Odense: Odense University Press; 1999. p. 145-72.

Herr M., Jeune B., Fors S., Andersen-Ranberg K., Ankri J., Arai Y., et al. Frailty and Associated Factors among Centenarians in the 5-COOP Countries. Gerontology. 2018. Jul 20:1-11.

Maier H., Gampe J., Jeune B., Robine J.M., Vaupel J.W., editors. Supercentenarians. Berlin: Berlin Heidelberg: Springer-Verlag; 2010.

2019 World Sickle Cell Awareness Day

Red blood cells, sickle shaped (Sickle Cell Disease) ©Inserm/Chevance de Boisfleury, Anne-Marie

Wednesday 19 June 2019 is World Sickle Cell Disease Day.

This is the most widespread genetic disorder in the world: It affects over five million people(1). It can have serious consequences—anaemia, episodes of pain—and may involve different organs or reduced resistance to certain infections.

From the physiological point of view, this disease of the blood is characterised by altered haemoglobin. Haemoglobin is the main constituent of the red blood cell. It is responsible for oxygen transport in the bloodstream, and its distribution to all the organs. The red blood cell becomes deformed, assumes a sickle shape (see illustration), and can no longer circulate through the blood vessels.

Research on sickle cell disease is currently aimed at improving treatments, by gene therapy and other means. Researchers are also studying the molecular mechanisms of the disease and the behaviour of abnormal haemoglobin.

Source (1): orphanet.fr

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