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Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy.
J Muscle Res Cell Motil
January 2025
Institute of Developmental and Regenerative Medicine, University of Oxford, IMS-Tetsuya Nakamura Building, Old Road Campus, Roosevelt Dr, Headington, Oxford, OX3 7TY, UK.
Recent years have seen enormous progress in the field of advanced therapeutics for the progressive muscle wasting disease Duchenne muscular dystrophy (DMD). In particular, four antisense oligonucleotide (ASO) therapies targeting various DMD-causing mutations have achieved FDA approval, marking major milestones in the treatment of this disease. These compounds are designed to induce alternative splicing events that restore the translation reading frame of the dystrophin gene, leading to the generation of internally-deleted, but mostly functional, pseudodystrophin proteins with the potential to compensate for the genetic loss of dystrophin.
View Article and Find Full Text PDFA comprehensive insights of cancer immunotherapy resistance.
Med Oncol
January 2025
Department of In Vivo Pharmacology, TCG Lifesciences Pvt. Ltd, BN 7, Sector V, Salt Lake City, Kolkata, West Bengal, 700091, India.
Cancer is a major global health issue that is usually treated with multiple therapies, such as chemotherapy and targeted therapies like immunotherapy. Immunotherapy is a new and alternative approach to treating various types of cancer that are difficult to treat with other methods. Although immune checkpoint inhibitors have shown promise for long-term efficacy, they have limited effectiveness in common cancer types such as breast, prostate, and lung.
View Article and Find Full Text PDFLowering LDL cholesterol by PCSK9 inhibition: a new era of gene silencing, RNA, and alternative therapies.
Naunyn Schmiedebergs Arch Pharmacol
January 2025
Institute of Pharmacy, Nirma University, Gujarat, 382481, India.
Proprotein convertase subtilisin/kexin type 9 (PCSK9) discovery has added a new paradigm to our understanding of cholesterol homeostasis and lipid metabolism. Since its discovery, PCSK9 inhibitors have become a widely investigated therapeutic class for lipid management in cardiovascular diseases and hypercholesterolemia. Scientists have explored different approaches for PCSK9 inhibition, such as monoclonal antibodies (mAbs), gene silencing and gene editing techniques, vaccines, mimetic peptides, and small molecules.
View Article and Find Full Text PDFThe role of green synthesis metal and metal oxide nanoparticles in oral cancer therapy: a review.
J Drug Target
January 2025
School of Stomatology, Wannan Medical College, Wuhu 241000, Anhui, China.
There are 275,000 new cases of oral cancer (OC) per year, making it the sixth most common cancer in the world. Severe adverse effects, including loss of function, deformity, and systemic toxicity, are familiar with traditional therapies such as radiation, chemotherapy, and surgery; due to their unique properties, nanoparticles (NPs) have emerged as a superior alternative over chemo/radiotherapy and surgery due to their targeting capability, bioavailability, compatibility, and high solubility. Due to their unique properties, metallic NPs have garnered significant attention in OC control.
View Article and Find Full Text PDFLong-Term Intracranial Progression-Free Survival in Lung Cancer Brain Metastases Extended Beyond 50 Months with Gubenxiaoyi Formula (GBXY): A Groundbreaking Case Study.
Integr Cancer Ther
January 2025
Department of Oncology, Beijing Hospital of Traditional Chinese Medicine, Capital Medical University, Beijing, China.
Background: The prevalence of brain metastases (BM) in lung cancer patients is notably high and is associated with poor prognoses. The efficacy of standard treatment regimens in improving intracranial progression-free survival (IPFS) for lung cancer BM is markedly limited. While traditional Chinese medicine (TCM) has been effective in enhancing the quality of life and prognosis of lung cancer patients, its efficacy in treating BM remains unreported.
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