CRISPR Therapeutics and Vertex announce positive safety and efficacy data from first two patients treated with CRISPR/Cas9 gene-editing therapy for severe hemoglobinopathies.
Link to the press release
PRESS RELEASE OCT. 4. 2018
Congenital genetic diseases can cause the immune system to malfunction, resulting in an absent defense mechanism against the microorganisms we are constantly exposed to. With the support of the Innovation Fund Denmark, medical doctors and researchers from Aarhus University Hospital and Aarhus University will develop new technology to correct these gene errors.
LIFE-THREATENING CONGENITAL DISEASES WILL IN THE FUTURE BE CURED BY GENE THERAPY. CONGENITAL GENETIC DEFECTS CAN RESULT IN THE IMMUNE SYSTEM FAILING TO DEFEND THE BODY AGAINST THE MICROORGANISMS WE ARE CONSTANTLY EXPOSED TO.
With the support of the Innovation Fund Denmark, researchers from Aarhus University aim to develop new technology to correct these defects
PRESS RELEASE: 03.10.2018 (innovationsfonden.dk)
Some of the most severe congenital diseases involve the immune system. When the immune system is not functioning, patients often die in the early years due to simple infections.
Knowledge of the genetic background of these diseases is now so advanced, allowing the exact genetic mutations to be identified.
With an investment of 25 million DKK from the Innovation Fund Denmark, researchers from Aarhus University will continue to develop the revolutionary CRISPR / Cas technique, which over the past 5 years has enabled accurate and efficient human genome editing.
The goal is to cure the diseases by correcting the genetic information in the patient's own stem cells that give rise to the immune system. - This project will eventually help children with rare immune defects to a new life. Children, who often do not survive to adulthood today, says Staff Specialist and Professor, Trine H. Mogensen from the Department of Infectious Medicine and the Department of Biomedicine, Aarhus University Hospital.
Professor at the Department of Clinical Medicine, Peter Hokland, who has been researching stem cells for over 30 years, adds: - With this support, a strong research team from Aarhus University and Aarhus University Hospital will be able to create a roadmap for the clinical introduction of CRISPR /Cas technique. We hope that before the end of the first 5-year period, we will be able to apply for and receive permission to treat the first patients. However, this will only be the first step in a multi-stage rocket, where other diseases caused by genetic defects can be quickly treated thereafter. This will take place in new and specialized laboratory facilities, which are being constructed by the Aarhus University, with a physical location at the hospital grounds in Skejby.
The "molecular scissors" called CRISPR is on everyone's lips. But what exactly can we use this hyped gene technology to accomplish? And how long do we have to wait? Get answers in this week's podcast.
According to biomedical associate professor Rasmus Bak, one of the problems that gene technology can help solve within in a number of years, is nothing less than cancer:
"I believe this is the field in which CRISPR will be implemented in clinical trials within two to three years – that is what I anticipate" says Rasmus Bak, Associate Professor at Department of Biomedicine and Aarhus Institute of Advanced Studies at Aarhus University, in the podcast.
CRISPR/ Cas9 is the name of a new and revolutionizing gene technology that has the potential to change mankind. Hopefully, continuous updates on the progress of the technology will pave the way for transparency, professor Peter Hokland writes.