Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1038/jid.2009.33 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Lentiviral Vector-Mediated Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model.
Hum Gene Ther
November 2024
Skeletal Dysplasia Research Lab, Nemours Children's Health, Wilmington, Delaware, USA.
Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disease caused by a mutation in the N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) gene resulting in progressive systemic skeletal dysplasia. There is currently no effective treatment available for this skeletal condition. Thus, the development of a new therapy stands as an unmet challenge in reversing or alleviating the progression of the disease.
View Article and Find Full Text PDFPOU2F2 activates the Akt/mTOR signalling pathway and enhances B lymphocyte function during diabetic kidney disease by promoting PIK3CD transcription.
Nephrology (Carlton)
December 2024
Department of Endocrinology, the Fourth Affiliated Hospital of Harbin Medical University, Harbin, Heilongjiang, People's Republic of China.
Background: Phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit delta (PIK3CD) is the predominant isoform of the catalytic subunit of PI3K in lymphocytes. Based on comprehensive bioinformatics, this study explores the functions of PIK3CD in diabetic kidney disease (DKD) progression in mice and B lymphocyte activity.
Methods: Non-obese diabetic (NOD) mice that spontaneously develop DKD were applied as animal models.
Low frequency‑pulsed electromagnetic fields promote osteogenic differentiation of bone marrow‑derived mesenchymal stem cells by regulating connexin 43 expression.
Exp Ther Med
December 2024
Department of Spine Surgery, Ganzhou People's Hospital (The Affiliated Ganzhou Hospital of Jiangxi Medical College of Nanchang University, Ganzhou Hospital-Nanfang Hospital of Southern Medical University), Ganzhou, Jiangxi 341000, P.R. China.
Article Synopsis
- - The study explored how connexin 43 (Cx43) influences the bone-forming ability of rat bone marrow-derived mesenchymal stem cells (BMSCs) when exposed to low-frequency-pulsed electromagnetic fields (LPEMF).
- - BMSCs were treated with LPEMF at 80 Hz for 1 hour, which led to increased cell proliferation and enhanced osteogenic markers, indicating improved bone formation capabilities.
- - Inhibiting Cx43 expression reduced the benefits seen from LPEMF, suggesting that Cx43 plays a crucial role in promoting osteogenic differentiation in BMSCs.
Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects.
Gene Ther
November 2024
Wake Forest Institute for Regenerative Medicine, Wake Forest University School of Medicine, Winston-Salem, NC, USA.
Article Synopsis
- Dent disease type 1 is linked to issues with the CLCN5 gene on the X chromosome, leading to problems like protein in urine and high calcium levels.
- Previous attempts at gene therapy showed short-term success in mice, likely hampered by immune responses against the ClC-5 protein.
- New strategies focused on targeting kidney cells and treating newborn mice show promise, suggesting that early intervention could improve outcomes and reduce immune rejection in patients with Dent disease type 1.
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
Mol Ther
November 2024
AVROBIO, Inc., Cambridge, MA 02139, USA; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Center, VU University, and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1081 HV, Amsterdam, the Netherlands; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, 1081 HV, Amsterdam, the Netherlands. Electronic address:
Article Synopsis
- Pompe disease is a rare genetic disorder caused by a deficiency in the enzyme acid alpha-glucosidase, resulting in muscle weakness due to glycogen accumulation in lysosomes.
- Enzyme replacement therapy (ERT) is the current standard treatment but has limitations, like poor muscle penetration and immune reactions against the therapy.
- This study explores a new treatment approach using lentiviral vector-mediated gene therapy in stem cells, showing promise in reversing the disease's effects in a mouse model, along with safety assessments and insights into the treatment's mechanisms.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!