[HTML][HTML] Lysosomal functions of progranulin and implications for treatment of frontotemporal dementia
MJ Simon, T Logan, SL DeVos, G Di Paolo - Trends in Cell Biology, 2023 - cell.com
Loss-of-function heterozygous mutations in GRN, the gene encoding progranulin (PGRN),
were identified in patients with frontotemporal lobar degeneration (FTLD) almost two …
were identified in patients with frontotemporal lobar degeneration (FTLD) almost two …
[HTML][HTML] Progranulin as a therapeutic target in neurodegenerative diseases
Progranulin (PGRN, encoded by the GRN gene) plays a key role in the development,
survival, function, and maintenance of neurons and microglia in the mammalian brain. It …
survival, function, and maintenance of neurons and microglia in the mammalian brain. It …
[HTML][HTML] An update on genetic frontotemporal dementia
CV Greaves, JD Rohrer - Journal of neurology, 2019 - Springer
Frontotemporal dementia (FTD) is a highly heritable group of neurodegenerative disorders,
with around 30% of patients having a strong family history. The majority of that heritability is …
with around 30% of patients having a strong family history. The majority of that heritability is …
Promoting the clearance of neurotoxic proteins in neurodegenerative disorders of ageing
Neurodegenerative disorders of ageing (NDAs) such as Alzheimer disease, Parkinson
disease, frontotemporal dementia, Huntington disease and amyotrophic lateral sclerosis …
disease, frontotemporal dementia, Huntington disease and amyotrophic lateral sclerosis …
Gene therapy for neurological disorders: progress and prospects
Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …
Neuroinflammation in frontotemporal dementia
Frontotemporal dementia (FTD) refers to a group of progressive neurodegenerative
disorders with different pathological signatures, genetic variability and complex disease …
disorders with different pathological signatures, genetic variability and complex disease …
Hypoxia‐stimulated ATM activation regulates autophagy‐associated exosome release from cancer‐associated fibroblasts to promote cancer cell invasion
L Xi, M Peng, S Liu, Y Liu, X Wan, Y Hou… - Journal of …, 2021 - Wiley Online Library
Cancer‐associated fibroblasts (CAFs) as a predominant cell component in the tumour
microenvironment (TME) play an essential role in tumour progression. Our earlier studies …
microenvironment (TME) play an essential role in tumour progression. Our earlier studies …
[HTML][HTML] Current and future prospects for gene therapy for rare genetic diseases affecting the brain and spinal cord
TL Jensen, CR Gøtzsche… - Frontiers in molecular …, 2021 - frontiersin.org
In recent years, gene therapy has been raising hopes towards viable treatment strategy for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …
rare genetic diseases for which there has been almost exclusively supportive treatment. We …
Lysosomal dysfunction at the centre of Parkinson's disease and frontotemporal dementia/amyotrophic lateral sclerosis
Parkinson's disease (PD) and frontotemporal dementia/amyotrophic lateral sclerosis
(FTD/ALS) are insidious and incurable neurodegenerative diseases that represent a …
(FTD/ALS) are insidious and incurable neurodegenerative diseases that represent a …
[HTML][HTML] Current and emerging treatment strategies for neuronal ceroid lipofuscinoses
A Kohlschütter, A Schulz, U Bartsch, S Storch - CNS drugs, 2019 - Springer
The neuronal ceroid lipofuscinoses comprise a group of neurodegenerative lysosomal
storage disorders caused by mutations in at least 13 different genes and primarily affect the …
storage disorders caused by mutations in at least 13 different genes and primarily affect the …