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GsMYB10 encoding a MYB-CC transcription factor enhances the tolerance to acidic aluminum stress in soybean.
BMC Plant Biol
December 2024
Key Laboratory of Plant Molecular Breeding of Guangdong Province, College of Agriculture, South China Agricultural University, Guangzhou, Guangdong, 510642, China.
Background: MYB transcription factors (TFs) play crucial roles in the response to diverse abiotic and biotic stress factors in plants. In this study, the GsMYB10 gene encoding a MYB-CC transcription factor was cloned from wild soybean BW69 line. However, there is less report on the aluminum (Al)-tolerant gene in this subfamily.
View Article and Find Full Text PDFComparative assessment of the transduction efficiency and safety associated with the delivery of AAV9-GFP vector via lumbar puncture to cynomolgus macaques with and without anti-AAV9 pre-existing antibodies.
Mol Ther Methods Clin Dev
December 2024
Preclinical Safety (PCS), Novartis Biomedical Research, Cambridge, MA, USA.
Administration of AAV-based gene therapies into the intra-cerebrospinal fluid (CSF) compartments via routes such as lumbar puncture (LP) has been implemented as an alternative to intravenous dosing to target the CNS regions. This route enables lower doses, decreases systemic toxicity, and circumvents intravascular pre-existing anti-AAV antibodies. In this study, AAV9-GFP vectors were administered via LP to juvenile cynomolgus macaques with and without pre-existing serum anti-AAV9 antibodies at a 5.
View Article and Find Full Text PDFA phase 1/2 safety and efficacy study of TAK-754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials.
Haemophilia
December 2024
Investigative Toxicology, Takeda Development Center of the Americas, Cambridge, USA.
Introduction: Haemophilia A is an X-linked bleeding disorder resulting from a deficiency of factor VIII (FVIII). To date, multiple gene therapies have entered clinical trials with the goal of providing durable haemostatic protection from a single dose. TAK 754 (BAX 888) is an investigational AAV8-based gene therapy containing a FVIII transgene.
View Article and Find Full Text PDFAccumulated BCAAs and BCKAs contribute to the HFD-induced deterioration of Alzheimer's disease via a dysfunctional TREM2-related reduction in microglial β-amyloid clearance.
J Neuroinflammation
December 2024
Department of Nutrition and Food Hygiene, Hubei Key Laboratory of Food Nutrition and Safety, MOE Key Laboratory of Environment and Health, School of Public Health, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, 430030, People's Republic of China.
A high-fat diet (HFD) induces obesity and insulin resistance, which may exacerbate amyloid-β peptide (Aβ) pathology during Alzheimer's disease (AD) progression. Branched-chain amino acids (BCAAs) accumulate in obese or insulin-resistant patients and animal models. However, roles of accumulated BCAAs and their metabolites, branched-chain keto acids (BCKAs), in the HFD-induced deterioration of AD and the underlying mechanisms remains largely unclear.
View Article and Find Full Text PDFCurrent and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy.
Hum Gene Ther
December 2024
Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
Adeno-associated virus (AAV) vectors have demonstrated safety and efficacy for gene transfer to hepatocytes in preclinical models, in various clinical trials and from a clinical experience with a growing number of approved gene therapy products. Although the exact duration is unknown, the expression of therapeutic genes in hepatocytes remains stable for several years after a single administration of the vector at clinically relevant doses in adult patients with hemophilia and other inherited metabolic disorders. However, clinical applications, especially for diseases requiring high AAV vector doses by intravenous administrations, have raised several concerns.
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