For patients

OVERVIEW OF GBA1 CANADA (G-CAN) INITIATIVE

BRAIN CELLS HAVE MULTIPLE GENES THAT PLAY A ROLE IN HOW THE BODY WORKS.

One of the genes called GBA1, contains information necessary to generate a protein called glucocerebrosidase (GCase). GCase is responsible for removing waste products in the brain cells. Thus, when there are changes in the DNA structure of the GBA1 gene, also known as mutations, waste products build up leading to brain cell death. This cell death impacts the proper functioning of the brain leading to diseases such as dementia with Lewy bodies (DLB) and Parkinson’s disease (PD). Having mutations in the GBA1 gene doesn’t guarantee that someone will get Parkinson’s, but it could make them more likely to develop it compared to someone without mutations. Therefore, it is crucial to study these gene mutations since there is currently no treatment that targets GBA1 mutations.

To achieve this goal, a GBA1 Canadian (G-Can) project has been initiated to conduct various research ideas to come up with GBA1 linked medication for treating these brain diseases. G-Can project brings together world-leading experts aiming to conduct research openly. This means that the research tools developed by this project will be freely accessible to anyone interested in furthering the research. In doing so, the discovery will also bring more awareness of these conditions not only to the research community but to the public.

Specifically, the project will utilize samples from patients (mixed populations) with GBA1 mutations and control group comprising of healthy individuals with the GBA1 mutations. Among the many tools that will be used by the G-Can project are the pluripotent stem cells. These cells, generated from patient samples, will be used to create any body cell/tissue allowing the scientists to study human development and causes of diseases in a controlled environment such as a culture dish. By using this approach, scientists can make models of different organs or tissues affected by these diseases. This then helps them observe how disease develops and eventually test potential treatments without the need to experiment directly on a person.

Through this project, it is anticipated that the research data, tools and treatments generated will ultimately save the lives of many suffering from GBA1 linked diseases and also further research in this field.

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