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Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
Neurology
January 2025
The Dubowitz Neuromuscular Centre, Developmental Neurosciences Department, University College London, Great Ormond Street Institute of Child Health, United Kingdom.
Background And Objectives: Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing <8.5 kg. SMART was the first clinical trial to evaluate onasemnogene abeparvovec for participants weighing 8.
View Article and Find Full Text PDFKorth toxicity: Experimental findings and future prospects.
J Taibah Univ Med Sci
December 2024
Drug Discovery and Synthetic Chemistry Research Group, Department of Pharmaceutical Chemistry, Kulliyyah of Pharmacy, International Islamic University Malaysia, Kuantan, Pahang, Malaysia.
(Roxb.) Korth, locally known as kratom, is a traditional medicinal plant from Southeast Asia, with mitragynine as its principal alkaloid. Similar to other medicinal plants, kratom has side effects and toxicities, which have been documented in scientific studies and case reports.
View Article and Find Full Text PDFEstrogens are key hormones that play a vital role in the physiology of the reproductive system in women. However, their therapeutic use in hormonal treatment, contraception, and the treatment of hormone-dependent diseases may be associated with a number of side effects, especially on the liver. This article focuses on the mechanisms of action of estrogens and their potential hepatotoxic effects, as well as risk factors and possible differences between representatives.
View Article and Find Full Text PDFAn engineered adeno-associated virus mediates efficient blood-brain barrier penetration with enhanced neurotropism and reduced hepatotropism.
J Control Release
January 2025
Wuhan National Laboratory for Optoelectronics, Huazhong University of Science and Technology, 430074 Wuhan, PR China; Shenzhen Key Laboratory of Viral Vectors for Biomedicine, Shenzhen-Hong Kong Institute of Brain Science, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, 518055 Shenzhen, PR China; Key Laboratory of Magnetic Resonance in Biological Systems, State Key Laboratory of Magnetic Resonance and Atomic and Molecular Physics, National Center for Magnetic Resonance in Wuhan, Innovation Academy for Precision Measurement Science and Technology, Chinese Academy of Sciences, 430071 Wuhan, PR China; Key Laboratory of Quality Control Technology for Virus-Based Therapeutics, Guangdong Provincial Medical Products Administration, NMPA Key Laboratory for Research and Evaluation of Viral Vector Technology in Cell and Gene Therapy Medicinal Products, The Brain Cognition and Brain Disease Institute, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, 518055 Shenzhen, PR China; University of Chinese Academy of Sciences, 100049 Beijing, PR China; Center for Excellence in Brain Science and Intelligence Technology, Chinese Academy of Sciences, 200031 Shanghai, PR China. Electronic address:
The blood-brain barrier (BBB) is a formidable barrier that restricts the entry of substances into the brain, complicating the study of brain function and the treatment of neurological conditions. Traditional methods of delivering genes from the periphery to the central nervous system (CNS) using adeno-associated viruses (AAVs) often require high doses, which can trigger immune responses and hepatotoxicity. Here, we developed a new AAV variant named AAVhu.
View Article and Find Full Text PDFDiscovering the compatibility of Coptidis Rhizoma and Gardeniae Fructus in attenuating hepatotoxicity through the association rules analysis of related formula.
J Ethnopharmacol
January 2025
Shenyang Medical College, Shenyang, Liaoning, China. Electronic address:
Ethnopharmacological Relevance: Gardenia jasminoides J. Ellis (Gardeniae Fructus, GF) is a widely used herbal medicine in many prescriptions. However, inappropriate application of GF may induce hepatotoxicity, which greatly challenges its clinical application.
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