Healing with “genetic scissors”, therapies with “tailor-made” cells and babies with “optimized” genes: what can genetic engineering methods achieve in medicine and what are the risks and ethical questions? In the June issue of Bild der Wissenschaft examines the topic of gene therapies in humans.
It is sequenced, synthesized and manipulated: Genetic processes have long shaped medical research and development in a variety of ways. But about ten years ago there was an almost revolutionary development in this area: Thanks to the “gene scissors” Crispr / Cas, geneticists can now intervene in the genome more specifically than ever before. Many people are concerned about the new ways of manipulating the genome – but this is offset by enormous medical potential: Specifically, the Crispr / Cas technology has raised high hopes in the fight against hereditary diseases, metabolic disorders or HIV / AIDS.
In the first article of the three-part title topic, the bdw author Rainer Kurlemann reports on the successes and developments in using the tool in these areas of application. For example, it has been possible to successfully treat hereditary sickle cell anemia with gene therapy based on the Crispr / Cas technology. The patient’s bone marrow cells are genetically “upgraded” in the laboratory and then used again. A similar approach could help people with HIV obtain immune cells that cannot be attacked by the Hi virus.
An eye on opportunities and risks
Both approaches are so-called ex vivo therapies in which the scientists carry out genetic engineering in the laboratory. But for other diseases, doctors use the Crispr / Cas gene scissors directly in the human body. There has also been progress in the development of these in vivo gene therapies – however, it is always important to keep an eye on the potential cancer risk, the author explains.
However, medical genetic engineering also has great potential in the fight against cancer, as the second part of the title topic shows. In it, the bdw author Gerlinde Felix reports how the so-called lymphatic leukemia can already be successfully combated by genetically engineered immune cells. The so-called CAR-T cell therapy also has the potential to treat other forms of cancer, explains the author. However, the process still has to mature in order to be able to use it against solid tumors, according to the article “Gene therapy against cancer”.
The cover story is rounded off by a look at the delicate subject of “designer babies from the laboratory”. In it, Rainer Kurlemann explains how targeted interventions in the germ line can theoretically prevent hereditary diseases. However, this involves health risks and difficult ethical questions. In this context, the author reports how, despite broad rejection, it has already happened that genetically modified babies have emerged. The version of highly intelligent genetic engineering people with superior traits, which seems creepy to many people, remains a fiction so far. One reason: characteristics such as intelligence are not based on individual genetic makeup, but on very complex genetic factors, reports Kurlemann.
You can find out more in the June issue of bild der Wissenschaft, which will be available in stores from May 18th.