Effects of lysosomal biotherapeutic recombinant protein expression on cell stress and protease and general host cell protein release in Chinese hamster ovary cells
Sign Up to like & getrecommendations! Published in 2017 at "Biotechnology Progress"
DOI: 10.1002/btpr.2455
Abstract: Recombinant human Acid Alpha Glucosidase (GAA) is the therapeutic enzyme used for the treatment of Pompe disease, a rare genetic disorder characterized by GAA deficiency in the cell lysosomes (Raben et al., Curr Mol Med.… read more here.
Keywords: protein; protease; gaa; cell ... See more keywords
Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease
Sign Up to like & getrecommendations! Published in 2017 at "Journal of Molecular Medicine"
DOI: 10.1007/s00109-017-1505-9
Abstract: Pompe disease is characterized by accumulation of both lysosomal and cytoplasmic glycogen primarily in skeletal and cardiac muscles. Mannose-6-phosphate receptor-mediated enzyme replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) targets the enzyme to lysosomes… read more here.
Keywords: antibody; glycogen; pompe disease; gaa ... See more keywords
Continued analysis of GAA mice treated with novel hybrid promoter rAAV vectors expressing acid alpha-glucosidase
Sign Up to like & getrecommendations! Published in 2019 at "Molecular Genetics and Metabolism"
DOI: 10.1016/j.ymgme.2018.12.168
Abstract: Pompe disease is caused by mutations in the acid alpha-glucosidase gene (GAA) that is responsible for processing lysosomal glycogen. Patients with Pompe disease exhibit clinical phenotypes across a variety of tissues, including glycogen buildup in… read more here.
Keywords: mice; muscle; gaa; raav vectors ... See more keywords
Identification of Gossypol Acetate as an Autophagy Modulator with Potent Anti-tumor Effect against Cancer Cells.
Sign Up to like & getrecommendations! Published in 2022 at "Journal of agricultural and food chemistry"
DOI: 10.1021/acs.jafc.1c06399
Abstract: Autophagy, an evolutionarily conserved process, is intricately involved in many aspects of human health and a variety of human diseases, including cancer. Discovery of small-molecule autophagy modulators with potent anticancer effect would be of great… read more here.
Keywords: cancer cells; effect; gaa; gossypol acetate ... See more keywords
Ganoderic Acid A Mitigates Inflammatory Bowel Disease through Modulation of AhR Activity by Microbial Tryptophan Metabolism.
Sign Up to like & getrecommendations! Published in 2024 at "Journal of agricultural and food chemistry"
DOI: 10.1021/acs.jafc.4c01166
Abstract: Inflammatory bowel disease (IBD), including Crohn's disease and ulcerative colitis, is a complex gastrointestinal condition influenced by genetic, microbial, and environmental factors, among which the gut microbiota plays a crucial role and has emerged as… read more here.
Keywords: disease; gaa; tryptophan metabolism; bowel disease ... See more keywords
Frequency of FGF14 intronic GAA repeat expansion in patients with multiple system atrophy and undiagnosed ataxia in the Japanese population
Sign Up to like & getrecommendations! Published in 2024 at "European Journal of Human Genetics"
DOI: 10.1038/s41431-024-01743-3
Abstract: Multiple system atrophy (MSA) is a neurodegenerative disorder characterized by autonomic nervous system dysfunction and cerebellar ataxia or parkinsonism. Recently, expanded GAA repeats (≥250 repeat units) in intron 1 of FGF14 have been shown to… read more here.
Keywords: repeat; system; gaa repeats; expanded gaa ... See more keywords
Performance, antioxidant status, nutrient retention and serum profile responses of laying Japanese quails to increasing addition levels of dietary guanidinoacetic acid
Sign Up to like & getrecommendations! Published in 2019 at "Italian Journal of Animal Science"
DOI: 10.1080/1828051x.2019.1698325
Abstract: Abstract In this study, the effects of different levels of dietary guanidinoacetic acid (GAA) supplementation (0, 0.6, 1.2 and 1.8 g/kg) in laying quails was evaluated in a completely randomised design comprised of four dietary treatments… read more here.
Keywords: observed gaa; performance; supplementation; antioxidant status ... See more keywords
Effects of dietary supplementation of guanidinoacetic acid on physiological response of broiler chicken exposed to repeated lactic acid injection
Sign Up to like & getrecommendations! Published in 2021 at "Italian Journal of Animal Science"
DOI: 10.1080/1828051x.2021.1873075
Abstract: Abstract Increased blood lactic acid is likely to be involved in the incidence of sudden death syndrome (SDS) in broiler chicken. Guanidinoacetic acid (GAA) with direct or indirect influence on the cardiovascular system may provide,… read more here.
Keywords: guanidinoacetic acid; broiler; broiler chicken; gaa ... See more keywords
Preparation and electrical performance optimization of SiGe channel FinFET and GAA transistors on SOI substrate
Sign Up to like & getrecommendations! Published in 2025 at "Semiconductor Science and Technology"
DOI: 10.1088/1361-6641/ade17e
Abstract: In this work, SiGe channel FinFET on SOI substrate is successfully prepared through a newly developed SiGe/Si-cap epitaxy and etching processes. This SiGe SOI FinFET demonstrates a high Ion of 3.6 × 10−4 A μm−1,… read more here.
Keywords: gaa; sige channel; channel finfet; soi substrate ... See more keywords
Disruption of the gaa Gene in Zebrafish Fails to Generate the Phenotype of Classical Pompe Disease.
Sign Up to like & getrecommendations! Published in 2017 at "DNA and cell biology"
DOI: 10.1089/dna.2016.3459
Abstract: The underlying pathogenic lesions of glycogen storage disease type II (GSD II) and the diversity of this disease among different species are still under exploration. Thus, we created an acid alpha-glucosidase (gaa) gene-mutated zebrafish model… read more here.
Keywords: disease; disruption gaa; gene zebrafish; gaa ... See more keywords
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
Sign Up to like & getrecommendations! Published in 2019 at "Human gene therapy"
DOI: 10.1089/hum.2018.016
Abstract: Pompe disease is an autosomal recessive glycogen storage disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). GAA deficiency results in systemic lysosomal glycogen accumulation and cellular disruption in muscle and the central… read more here.
Keywords: aavb1 gaa; gaa; glycogen; pompe disease ... See more keywords