{"id":771,"date":"2020-05-05T15:07:15","date_gmt":"2020-05-05T15:07:15","guid":{"rendered":"https:\/\/mightymaje.de\/?page_id=771"},"modified":"2022-06-16T09:45:19","modified_gmt":"2022-06-16T09:45:19","slug":"research","status":"publish","type":"page","link":"https:\/\/mightymaje.de\/en\/research","title":{"rendered":"Research"},"content":{"rendered":"\t\t
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Current research<\/h1>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Did you know that thanks to the latest developments in medical genetic engineering, some genetic diseases are treatable?
People with genetic sickle cell anemia now have the chance to live longer, carefree lives thanks to gene therapy tools.
Children with spinal muscular atrophy (SMA) can better maintain muscle strength and incredibly increase their independence thanks to gene therapy drugs. This has been the subject of recent debates about the allocation of these very expensive drugs and what life is worth to us as a society.<\/p>

In 2012, genetic engineering experienced a major breakthrough with the discovery of CRISPR\/Cas 9.<\/strong> CRISPR\/Cas 9 is a method that creates targeted mutations at a predetermined location in the genome to obtain desired characteristics. The molecular biology tool is also known as gene scissors<\/strong> or gene surgery.\u2028 The technique can be used to replace single base pairs in genetic material to correct a disease gene, for example. It can also be used to create a mutation at a predetermined site in the inheritance so that protein production from a particular gene is stopped, or to insert a gene at a specific site in the inheritance.<\/p>

Although the technology is now available, each genetic disorder must be considered separately and more research is needed before therapy for laminopathies is possible.<\/strong><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Research approaches Laminopathies<\/strong> <\/h3>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Research interest in laminopathies is not particularly high, but is growing due to the great commitment of affected families and associations and also thanks to new technical possibilities.
Various research projects on laminopathies are underway worldwide. For example, under the direction of Dr. Gis\u00e8le Bonne <\/a>in Paris. In Spain, Dr. Ignacio P\u00e9rez de Castro<\/a> is one of the leading researchers.
In both research institutes the Spanish foundation “FUNDACI\u00d3N ANDR\u00c9S MARCIO”<\/a> is involved. \u2028In Italy, the Association A.I.D.M.E.D.<\/a> is researching a therapy for Emery Dreyfuss.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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The Max Delbr\u00fcck Center in Berlin<\/strong><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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At the Max Delbr\u00fcck Center in Berlin, an institute of the Helmholtz Association and the Charit\u00e9, Prof. Dr. Simone Spuler<\/a> and her team have been working for a long time on the development of potential therapies for muscle diseases. The MDC is currently the greatest hope in Germany for a gene therapy for people with muscle diseases.<\/strong><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Maje in Berlin<\/h3>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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In June 2019, we met Dr. Spuler and her team in Berlin. She gave us a tour of her institute and convinced us of her research approach and experience. \u2028Therefore, we decided to fund a first LMNA project at the Max Delbr\u00fcck Center with the donations we have received so far.
The results could give hope to many affected people to stop the disease!<\/strong><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Maje with her brother in the laboratory of Prof. Dr. Simone Spuler<\/figcaption>\n\t\t\t\t\t\t\t\t\t\t<\/figure>\n\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Majes cells at Max Delbr\u00fcck Center<\/h3>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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What happened so far:<\/strong><\/p>