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Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
Front Neurol
January 2025
Department of Neurology, University of California, Irvine, Irvine, CA, United States.
[This corrects the article DOI: 10.3389/fneur.2024.
View Article and Find Full Text PDFCurrent approaches in CRISPR-Cas system for metabolic disorder.
Prog Mol Biol Transl Sci
January 2025
School of Health Sciences & Technology, UPES, Dehradun, Uttarakhand, India. Electronic address:
A new era in genomic medicine has been brought by the development of CRISPR-Cas technology, which presents hitherto unheard-of possibilities for the treatment of metabolic illnesses. The treatment approaches used in CRISPR/Cas9-mediated gene therapy, emphasize distribution techniques such as viral vectors and their use in preclinical models of metabolic diseases like hypercholesterolemia, glycogen storage diseases, and phenylketonuria. The relevance of high-throughput CRISPR screens for target identification in discovering new genes and pathways associated with metabolic dysfunctions is an important aspect of the discovery of new approaches.
View Article and Find Full Text PDFNeonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.
bioRxiv
December 2024
Department of Pediatrics, University of Florida, Gainesville, FL.
Absence of functional acid-α-glucosidase (GAA) leads to early-onset Pompe disease with cardiorespiratory and neuromuscular failure. A novel Pompe rat model ( ) was used to test the hypothesis that neonatal gene therapy with adeno-associated virus serotype 9 (AAV9) restores cardiorespiratory neuromuscular function across the lifespan. Temporal vein administration of AAV9-DES-GAA or sham (saline) injection was done on post-natal day 1; rats were studied at 6-12 months old.
View Article and Find Full Text PDFCorrection: Glycogen Synthase Kinase-3 regulates IGFBP-1 gene transcription through the Thymine-rich Insulin Response Element.
BMC Mol Cell Biol
December 2024
Department of Pathology and Neurosciences, University of Dundee, Ninewells Medical School and Hospital, Dundee, DD1 9SY, UK.
Sex-specific maladaptive responses to acute stress upon in utero THC exposure are mediated by dopamine.
Pharmacol Res
December 2024
Dept. Biomedical Sciences, Div. Neuroscience and Clinical Pharmacology, University of Cagliari, Italy. Electronic address:
Article Synopsis
- * Research demonstrates that male rats exposed to THC during pregnancy exhibit altered dopamine activity and increased vulnerability to stress, affecting their sensorimotor functions.
- * The study found that the HPA axis's regulation of dopamine neurons differs by sex, influencing stress responses and sensorimotor gating, with potential for therapeutic interventions to correct these disruptions.
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