Gene Therapy Cures Hemophilia


Hemophilia is a serious hereditary disease that prevents the blood from clotting. This means that, in the event of a wound, bleeding doesn’t stop or is extremely difficult to stop.

There are two types of hemophilia:

– Hemophilia A, the more common form, is linked to a deficiency in clotting factor VIII

– Hemophilia B, occurring more rarely, is linked to a deficiency in clotting factor IX

Hemophilia primarily affects boys and occurs very rarely in girls, since the disease is passed down on the X chromosome.

Two clinical studies recently published in The New England Journal of Medicine show that it is now possible to cure both hemophilia A and hemophilia B through gene therapy. A one-hour transfusion of a rescue gene that codes for the faulty clotting factor was enough to cure patients suffering from the disease. This is a breakthrough in treating the disease, and it is also one step further for gene therapy, which is being used more and more often in clinical trials.


French women breastfeed for 4 months on average

According to the World Health Organisation (WHO), exclusive breastfeeding (children only receiving breast milk and no other food or drink) should last until the infant is 6 months old and partial breastfeeding should continue up to 2 years of age. A study conducted by Inserm Unit 1018 “Centre for Research in Epidemiology and Population Health”, and published by the InVs (French Institute for Public Health Surveillance) Bulletin Épidémiologique Hebdomadaire, shows that French women breastfeed for an average of 17 weeks, or just over 4 and a half months in total, and only exclusively for two months.

These data come from the ELFE study organised by Inserm, Ined and the French National Blood Service, which monitored over 18,000 children since 2011. One of the aims was to analyse the duration for full and partial breastfeeding and then determine the sociocultural, demographical and economical factors associated with breastfeeding duration.

Researchers have demonstrated that if breastfeeding duration among women is shorter than the time recommended by WHO, then this observation is particularly true for women under 30 who live alone and have a low level of education.

For the authors of the study “These findings support the need to change the image of breastfeeding and to devise a promotional strategy that targets young mothers with low income and education that are more likely stop breastfeeding early”.

Best wishes 2014

Throughout the year, the press office filmed Inserm researchers talking about their discoveries in the simplest possible terms. The short “making of” below shows that this was not always easy. Enjoy the bloopers of 2013!

We at the Inserm press office hope that these 2 minutes will make you smile as much as they did us. And we wish you all the best for 2014.

To watch and share the video: and as always, to contact us, a single address: rf.mresni@esserp
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Out-of-hospital births and the supply of maternity units in France

Child abuse and denial

The verdict in the trial of the couple who killed their daughter Marina is due to be delivered on 27 June 2012. The trial has highlighted the blindness of many professionals, an issue raised by A. Tursz and C. Gréco in an editorial: “Child abuse: how do we overcome denial?” in the journal Archives de Pédiatrie.


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