New Gene Therapy Transplantation can Treat Neurodegenerative Diseases

As per the new study, a novel gene therapy transplantation technique developed can treat various neurodegenerative diseases. The genetically engineered immune cells are injected into brain ventricles can make therapeutic action faster. The central nervous system is defended by the Immune cells (the so-called microglia) have a key part in many neurodegenerative diseases.

‘Transplanting genetically engineered stem cells into stem cells ventricles can release molecules that help in tissue healing and regeneration and thereby, treating thereby neurodegenerative diseases.’

The research was performed for the first time at San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) with the collaboration of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. The research was published in Science Advances.

Alessandra Biffi, director of the Gene therapy program at Dana-Farber/Boston Children’s, the researchers have been guided. The technique has been tested on an experimental model for a metabolic disease and might have future therapeutic applications for other neurodegenerative diseases in which microglia cells play a role.

The research was supported also by the European Commission through an ERC give won by Alessandra Biffi. This research has its roots in Alessandra Biffi’s long-standing interest for lysosomial storage diseases (LSDs), a heterogeneous group of hereditary metabolic diseases caused by a genetic mutation preventing cells from producing enzymes which are fundamental for their metabolism. Patients with LSDs have serious damages to different organs and tissues, including the central nervous system.

The standard therapeutic approach, which is frequently the only one at hand comprises in the injection in the bloodstream of the missing enzyme. That, however, is incapable on the off chance that the patient has already shown neurological problems – in fact, the blood-brain barrier prevents the enzyme from reaching the brain, where neurodegeneration proceeds.

That is the reason a couple of years ago Alessandra Biffi, working with Luigi Naldini (director of SR-Tiget), tried cell and gene therapies approaches. In a continuous clinical trial performed at SR-Tiget to treat metachromatic leukodystrophy (a neurodegenerative LSD), blood stem cells are extracted from the patients’ bone marrow, genetically engineered so as they regain the ability to produce the missing enzyme, and after that injected back into the bloodstream. Having the ability to cross the blood-brain barrier, the cells reach the brain and start their therapeutic activity.

Timing, however, is a key component. Gene therapies administered through the blood are effective only on the off chance that they act in advance. Cells, in fact, require time to reach the cerebrum and engraft into the nervous tissue. That is the reason at the moment only patients who are as still asymptomatic can be treated. The new technique designed by Alessandra Biffi’s group and discussed on the today’s issue of Science Advances could change everything.

Alessandra Biffi. But the research results extend beyond the timing question says, “Transplanting stem cells into brain ventricles stimulates the engraftment procedure and later on could be a viable therapeutic option for patients demonstrating the first symptoms, as well”.

“Our information appears for the first time that transplanted blood stem cells, once they reach the brain, can differentiate into microglia-like cells. These cells remain only in the central nervous system”. This implies with such procedure we can rapidly generate a populace of genetically engineered cells only in the ill nervous system.

From their position, these cells can release therapeutic molecules, helping the tissue to heal and regenerate, and may have potential application in a variety of neurodegenerative diseases.


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