Gene Therapy in Parkinson's Disease
"We have advanced technology and cutting-edge techniques to implement strategies to investigate the origin and development of Parkinson's disease."
DR. JOSÉ LUIS LANCIEGO PÉREZ DIRECTOR. GENE THERAPY IN PARKINSON'S DISEASE RESEARCH GROUP
Parkinson's disease is characterized by the progressive death of nerve cells (neurons) that produce dopamine, a major chemical in the regulation of multiple brain functions involved in the control of movement.
It begins around the age of 60 but can appear earlier. It is the second most common neurodegenerative disease with a rising incidence and prevalence due to increased life expectancy.
There is no cure for this disease but there are some drugs or treatments that replenish the dopamine deficit and help to improve symptoms such as tremor, clumsiness, stiffness or other movement disorders.
The Parkinson's Disease Group at Cima is formed by clinical neurologists and expert researchers focused on finding the causes of this disease, still unknown, and developing new therapies capable of preventing the loss of dopamine and the extension of neurodegeneration to other brain cells.
To this end, we have implemented cutting-edge experimental parkinsonian models in the biomedical field that allow us to design innovative and effective strategies to address the treatment of the disease, such as the application of gene therapy.
Research Group Objectives
Abordamos el estudio de esta enfermedad y otros trastornos del movimiento con un enfoque traslacional: del laboratorio al paciente
To understand the causes and mechanisms of neuronal degeneration.
To find therapeutic targets that prevent or delay the development of the disease.
Search for biomarkers for early diagnosis and for monitoring the evolution of the disease.
Lines of research
We conduct experimental studies, in various animal models and cell cultures, and clinical studies in patients affected by Parkinson's disease or at risk of developing the disease or other movement disorders
Mutations in the GBA1 gene coding for a lysosomal enzyme called glucocerebrosidase are the main genetic risk factor for Parkinson's disease. These mutations cause the enzymatic activity of glucocerebrosidase to be greatly diminished, which is associated with accumulation and intracellular deposits of alpha-synuclein.
Studies of our group have shown that the use of viral vectors carrying the GBA1 gene can increase glucocerebrosidase levels and clear synuclein aggregates in both rodents and non-human primates, preventing cell death of dopaminergic neurons, decreasing the reactive response of microglial cells and minimizing the progression of alpha-synuclein pathology.
- Our work focuses on using glucocerebrosidase gene therapy to attenuate or even halt the progressive course that characterizes this neurodegenerative disease.
AND-PD European Consortium
Our laboratory participates in the European AND-PD (Comorbidity mechanisms of anxiety and Parkinson's disease) consortium, as part of a joint effort involving 11 European institutions, including universities, research centers and biotechnology-based companies.
The project aims to investigate the pathological mechanisms causing the anxiety and depression that are typically observed as comorbid entities in more than 40% of Parkinsonian patients.
We are developing new animal models using gene therapy tools. These models reproduce the neuropathological findings that typically characterize Parkinson's disease with unprecedented accuracy. Such models are critical in understanding the mechanisms underlying the progressive loss of dopamine-producing cells in the brain.
A wide variety of treatments are currently available for Parkinson's disease, although these treatments only alleviate the symptoms but do not cure the disease, which is progressive in nature. We have several gene therapy projects with different candidate genes designed to slow or even halt the progressive course of this neurodegenerative disease.
Meet the research team
Scientific activity of the
Parkinson's Disease Research Group
Latest scientific publications
- Development and characterization of a non-human primate model of disseminated synucleinopathy Dec 15, 2023 | Magazine: BiorXiv
- Neuromelanin accumulation drives endogenous synucleinopathy in non-human primates Dec 1, 2023 | Magazine: Brain
- Boolean analysis shows a high proportion of dopamine D2 receptors interacting with adenosine A2A receptors in striatal medium spiny neurons of mouse and non-human primate models of Parkinson's disease Nov 15, 2023 | Magazine: Neurobiology of Disease
- Preferential expression of SCN1A in GABAergic neurons improves survival and epileptic phenotype in a mouse model of Dravet syndrome Oct 11, 2023 | Magazine: Journal of Molecular Medicine