Big News from the Charité

Dear supporters of Maje, we wish you a happy new year 2021! Better than the last one and above all with more TOGEHTER and less hurdles! We start optimistically into the new year, because since last week we know that there are first indications of a gene correction on Maje's cells in the Berlin research laboratory of the Charité! ????????

A year ago, Dr. Wang started working on a strategy to correct Maje’s gene mutation. Since then, we have been getting regular glimpses of her work. The genetic cause of Maje’s disease is solely due to an amino acid that should not be where it is (“point mutation”). The researcher’s goal is to correct this “wrong” amino acid and change it into the correct one.

Using a so-called gene pincette (similar to gene scissors), Dr. Wang succeeded in correcting the “wrong” amino acid for the first time! This is a very important step.

Of course, there is still a long way to go before these strategies can one day be applied to humans.
But thanks to you and all the support we receive and the wonderful team of Dr. Spuler, we are confident.

Also, we have Maje. With her, anything is possible! 🦸🏻 Please continue to keep your fingers crossed for us 💕

The photo: (Attention now it's getting scientific 😉) The human induced pluripotent stem cells (iPSC) come from Maje's blood. The mutant lamin A / C (green) is shown on the nuclear envelope. Images were taken with a Zeiss LSM confocal microscope. © Dr. Haicui Wang and Prof. Dr. Simone Spuler, MDC and Charité, Berlin