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An international research team led by the University of Padua and the VIB-VUB Center for Structural Biology in Brussels has identified a type of antibody that can improve the function of a key protein in Parkinson's disease. The study, published in «Nature Communications», opens new therapeutic prospects for this neurodegenerative disease.
The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
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"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
The researchers have thus developed nanobodies that stabilise or activate glucocerebrosidase without blocking its active site, significantly improving the enzyme's function in cellular models. This approach could lead to new therapies for Parkinson's patients. Despite the preliminary nature of the results, the research team, according to Chiara Sinisgalli, the first author of the article, "will continue to develop methods to deliver these nanobodies to damaged brain cells, with the goal of transforming these discoveries into innovative therapeutic strategies."
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The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
Parkinson's disease is the second most common neurodegenerative disease, affecting around 10 million people worldwide. Initial symptoms include tremors and movement issues that worsen over time, leading to rigidity, slowness, balance problems, and in advanced stages, dementia. The exact causes of the disease remain unclear, but it is believed to be a mix of genetic and environmental factors. The malfunction of the enzyme glucocerebrosidase, responsible for the degradation of certain classes of lipids in cells, is one of the major risk factors.
"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
The researchers have thus developed nanobodies that stabilise or activate glucocerebrosidase without blocking its active site, significantly improving the enzyme's function in cellular models. This approach could lead to new therapies for Parkinson's patients. Despite the preliminary nature of the results, the research team, according to Chiara Sinisgalli, the first author of the article, "will continue to develop methods to deliver these nanobodies to damaged brain cells, with the goal of transforming these discoveries into innovative therapeutic strategies."
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An international research team led by the University of Padua and the VIB-VUB Center for Structural Biology in Brussels has identified a type of antibody that can improve the function of a key protein in Parkinson's disease. The study, published in «Nature Communications», opens new therapeutic prospects for this neurodegenerative disease.
The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
Parkinson's disease is the second most common neurodegenerative disease, affecting around 10 million people worldwide. Initial symptoms include tremors and movement issues that worsen over time, leading to rigidity, slowness, balance problems, and in advanced stages, dementia. The exact causes of the disease remain unclear, but it is believed to be a mix of genetic and environmental factors. The malfunction of the enzyme glucocerebrosidase, responsible for the degradation of certain classes of lipids in cells, is one of the major risk factors.
"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
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The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
Parkinson's disease is the second most common neurodegenerative disease, affecting around 10 million people worldwide. Initial symptoms include tremors and movement issues that worsen over time, leading to rigidity, slowness, balance problems, and in advanced stages, dementia. The exact causes of the disease remain unclear, but it is believed to be a mix of genetic and environmental factors. The malfunction of the enzyme glucocerebrosidase, responsible for the degradation of certain classes of lipids in cells, is one of the major risk factors.
"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
The researchers have thus developed nanobodies that stabilise or activate glucocerebrosidase without blocking its active site, significantly improving the enzyme's function in cellular models. This approach could lead to new therapies for Parkinson's patients. Despite the preliminary nature of the results, the research team, according to Chiara Sinisgalli, the first author of the article, "will continue to develop methods to deliver these nanobodies to damaged brain cells, with the goal of transforming these discoveries into innovative therapeutic strategies."
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The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
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"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
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The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
Parkinson's disease is the second most common neurodegenerative disease, affecting around 10 million people worldwide. Initial symptoms include tremors and movement issues that worsen over time, leading to rigidity, slowness, balance problems, and in advanced stages, dementia. The exact causes of the disease remain unclear, but it is believed to be a mix of genetic and environmental factors. The malfunction of the enzyme glucocerebrosidase, responsible for the degradation of certain classes of lipids in cells, is one of the major risk factors.
"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
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The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
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"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
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The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
Parkinson's disease is the second most common neurodegenerative disease, affecting around 10 million people worldwide. Initial symptoms include tremors and movement issues that worsen over time, leading to rigidity, slowness, balance problems, and in advanced stages, dementia. The exact causes of the disease remain unclear, but it is believed to be a mix of genetic and environmental factors. The malfunction of the enzyme glucocerebrosidase, responsible for the degradation of certain classes of lipids in cells, is one of the major risk factors.
"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
The researchers have thus developed nanobodies that stabilise or activate glucocerebrosidase without blocking its active site, significantly improving the enzyme's function in cellular models. This approach could lead to new therapies for Parkinson's patients. Despite the preliminary nature of the results, the research team, according to Chiara Sinisgalli, the first author of the article, "will continue to develop methods to deliver these nanobodies to damaged brain cells, with the goal of transforming these discoveries into innovative therapeutic strategies."
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An international research team led by the University of Padua and the VIB-VUB Center for Structural Biology in Brussels has identified a type of antibody that can improve the function of a key protein in Parkinson's disease. The study, published in «Nature Communications», opens new therapeutic prospects for this neurodegenerative disease.
The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
Parkinson's disease is the second most common neurodegenerative disease, affecting around 10 million people worldwide. Initial symptoms include tremors and movement issues that worsen over time, leading to rigidity, slowness, balance problems, and in advanced stages, dementia. The exact causes of the disease remain unclear, but it is believed to be a mix of genetic and environmental factors. The malfunction of the enzyme glucocerebrosidase, responsible for the degradation of certain classes of lipids in cells, is one of the major risk factors.
"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
The researchers have thus developed nanobodies that stabilise or activate glucocerebrosidase without blocking its active site, significantly improving the enzyme's function in cellular models. This approach could lead to new therapies for Parkinson's patients. Despite the preliminary nature of the results, the research team, according to Chiara Sinisgalli, the first author of the article, "will continue to develop methods to deliver these nanobodies to damaged brain cells, with the goal of transforming these discoveries into innovative therapeutic strategies."
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An international research team led by the University of Padua and the VIB-VUB Center for Structural Biology in Brussels has identified a type of antibody that can improve the function of a key protein in Parkinson's disease. The study, published in «Nature Communications», opens new therapeutic prospects for this neurodegenerative disease.
The research focuses on the enzyme glucocerebrosidase, the loss of function of which is linked to Parkinson's disease. When the enzyme does not function properly, brain cells gradually lose their normal activity. The special antibodies, called nanobodies, could reverse this process, restoring the enzyme's activity and cellular metabolism.
Parkinson's disease is the second most common neurodegenerative disease, affecting around 10 million people worldwide. Initial symptoms include tremors and movement issues that worsen over time, leading to rigidity, slowness, balance problems, and in advanced stages, dementia. The exact causes of the disease remain unclear, but it is believed to be a mix of genetic and environmental factors. The malfunction of the enzyme glucocerebrosidase, responsible for the degradation of certain classes of lipids in cells, is one of the major risk factors.
"A way to restore glucocerebrosidase function is to stabilise or activate it using so-called 'molecular chaperones', which are molecules capable of binding glucocerebrosidase," explains Nicoletta Plotegher, a lecturer at the Department of Biology at the University of Padua and corresponding author of the article. "However, most existing chaperones unfortunately bind to the enzyme's active site, partially blocking its activity, which greatly limits their effectiveness. We have developed an entirely new approach to improve glucocerebrosidase function using 'nanobodies', which are small fragments of special antibodies produced by camelids. Specifically, thanks to funding from the Michael J. Fox Foundation, we have identified nanobodies capable of stabilising or activating glucocerebrosidase by binding to regions of the enzyme far from the active site."
The researchers have thus developed nanobodies that stabilise or activate glucocerebrosidase without blocking its active site, significantly improving the enzyme's function in cellular models. This approach could lead to new therapies for Parkinson's patients. Despite the preliminary nature of the results, the research team, according to Chiara Sinisgalli, the first author of the article, "will continue to develop methods to deliver these nanobodies to damaged brain cells, with the goal of transforming these discoveries into innovative therapeutic strategies."
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