Babylieben saved: First personalized gene therapy in the USA!

Babylieben saved: First personalized gene therapy in the USA!

In the USA, a pioneering step in the treatment of rare hereditary diseases was taken. For the first time, a nine and a half months old baby, KJ Muldoon, received a personalized genome editing due to his life-threatening state, known as CPS1 deficit. This rare genetic disease affects a vital liver enzyme that is responsible for the breakdown of toxic metabolic products, which can lead to violent health problems without treatment. The therapy, which was coordinated on the genetic variants of KJ, could save his life after the parents were informed about the dark forecast shortly after the diagnosis, as [Kosmo] (https://www.kosmo.at/baby-mit-to-toticer-erbsteit-kommt-erstal- mass-tailed-gent therapy/).

Innovative treatment uses the CrisPR-CAS9 technology, which was awarded the Chemical Nobel Prize in 2020. This method is a genetic scissor that has the potential to heal genetic diseases by doing precisely DNA modifications. The treating doctors, including the doctor Rebecca Ahrens-Nicklas, were convinced that this therapy, which was never previously applied, could be hope for KJ. In February 2025, the baby received the first infusion of the tailor -made medication, followed by two further treatment. After therapy, KJ has already made progress; It can now eat more protein food and needs fewer medication, such as T-Online supplemented.

progress and challenges in gene therapy

The application of CrisPR-CAS9 represents significant progress in the targeted treatment of genetic disorders. This technology, which originally emerged from bacterial defense mechanisms, allows precise modifications of the genetic engineering and has already shown success in other areas, such as the treatment of blood diseases or cystic fibrosis. However, the long -term security and effectiveness of these therapies are still the subject of ongoing research. A comprehensive follow -up of KJ and similar patients will be necessary to make concrete statements about the treatment results.

in connection with the development of such personalized therapies there are also ethical considerations. The cost of such treatments is in the multi -digit million range, which severely limits access. So far, no comparable personalized gene therapy has been approved in the European Union, and experts in Austria urgently call for a debate about access to these innovative treatments and the ethical questions associated with it. This is particularly evident in the discussion about the responsibility associated with the application of crispr technologies, as mentioned in an overview of the challenges and future perspectives of the Crispr-Cas9 technology (pmc).

The treatment of KJ Muldoon could possibly pave the way for further innovative therapies and gives hope not only for affected families, but also for medical research as a whole. The coming time will show how the results of this first personalized gene therapy develop and what effects you will have on future treatment options.