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Givosiran: a targeted treatment for acute intermittent porphyria.
Hematology Am Soc Hematol Educ Program
December 2024
Harvard Medical School, Boston, MA.
The acute hepatic porphyrias (AHPs) are a family of rare genetic diseases associated with attacks of abdominal pain, vomiting, weakness, neuropathy, and other neurovisceral symptoms. Pathogenic variants in 1 of 4 enzymes of heme synthesis are necessary for the development of AHP, and the onset of acute attacks also requires the induction of δ-aminolevulinic acid synthase 1 (ALAS1), the first and rate-limiting step of heme synthesis in the liver. Givosiran is an RNA interference medication that inhibits hepatic ALAS1 and was designed to treat AHP.
View Article and Find Full Text PDFRenal and Vascular Functional Decline in Aged Low Birth Weight Murine Adults.
Kidney Blood Press Res
December 2024
Department of Medicine, New York Medical College, Valhalla, New York, USA.
Article Synopsis
- Maternal undernutrition leads to low birth weight in neonates, which can increase the risk for high blood pressure and kidney disease later in life, though the exact reasons are not fully understood.
- The study utilized a mouse model to examine factors like oxidative stress and vascular function in low birth weight mice after one year, measuring various health indicators and testing different pharmacological treatments.
- Results showed that low birth weight mice had higher blood pressure and decreased kidney function, but administration of specific drugs improved their conditions, indicating that therapies targeting oxidative stress and vascular function might mitigate some long-term health issues.
Protecting Against Postsurgery Oral Cancer Recurrence with an Implantable Hydrogel Vaccine for In Situ Photoimmunotherapy.
Adv Sci (Weinh)
December 2024
Department of Oral and Maxillofacial-Head Neck Oncology, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, College of Stomatology, Shanghai Jiao Tong University, Shanghai, 200011, China.
Oral squamous cell carcinoma (OSCC) often recurs aggressively and metastasizes despite surgery and adjuvant therapy, driven by postoperative residual cancer cells near the primary tumor site. An implantable in situ vaccine hydrogel was designed to target residual OSCC cells post-tumor removal. This hydrogel serves as a reservoir for the sustained localized release of δ-aminolevulinic acid (δ-ALA), enhancing protoporphyrin IX-mediated photodynamic therapy (PDT), and a polydopamine-hyaluronic acid composite for photothermal therapy (PTT).
View Article and Find Full Text PDFStrigolactone and nitric oxide collaborate synergistically to boost tomato seedling resilience to arsenic toxicity via modulating physiology and antioxidant system.
Plant Physiol Biochem
February 2024
Department of Botany and Microbiology, College of Science, King Saud University, Riyadh, 11451, Saudi Arabia. Electronic address:
Arsenic (As) poses a significant environmental threat as a metalloid toxin, adversely affecting the health of both plants and animals. Strigolactones (SL) and nitric oxide (NO) are known to play crucial roles in plant physiology. Therefore, the present experiment was designed to investigate the potential cumulative role of SL (GR24-0.
View Article and Find Full Text PDFSub-lethal impacts of lead poisoning on blood biochemistry, immune function and delta-aminolevulinic acid dehydratase (δ-ALAD) activity in Cape (Gyps coprotheres) and white-backed (G. africanus) Vulture chicks.
Environ Res
March 2024
DSI-NRF Centre of Excellence at the FitzPatrick Institute, Department of Zoology and Entomology, University of Pretoria, Pretoria, South Africa; South African Research Chair in Conservation Physiology, South African National Biodiversity Institute, Pretoria, South Africa.
Although the prevalence of lead poisoning in southern Africa's Gyps vultures is now well-established, its finer physiological effects on these endangered species remain poorly characterised. We evaluated the sub-lethal impact of acute lead exposure on Cape and White-backed Vulture chicks from two breeding colonies in South Africa, by analysing its possible effects on key blood biochemistry parameters, immune function, packed cell volume and δ-aminolevulinic acid dehydratase (δ-ALAD) activity. All 37 White-backed Vulture nestlings sampled displayed elevated lead levels (>10 μg/dL), and seven had blood [Pb] >100 μg/dL.
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