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Lipid-nanoparticle-enabled nucleic acid therapeutics for liver disorders
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作者 Porkizhi Arjunan Durga Kathirvelu +4 位作者 gokulnath mahalingam Ashish Kumar Goel Uday George Zacharaiah Alok Srivastava Srujan Marepally 《Acta Pharmaceutica Sinica B》 SCIE CAS CSCD 2024年第7期2885-2900,共16页
Inherited genetic disorders of the liver pose a significant public health burden.Liver transplantation is often limited by the availability of donor livers and the exorbitant costs of immunosuppressive therapy.To over... Inherited genetic disorders of the liver pose a significant public health burden.Liver transplantation is often limited by the availability of donor livers and the exorbitant costs of immunosuppressive therapy.To overcome these limitations,nucleic acid therapy provides a hopeful alternative that enables gene repair,gene supplementation,and gene silencing with suitable vectors.Though viral vectors are the most efficient and preferred for gene therapy,pre-existing immunity debilitating immune responses limit their use.As a potential alternative,lipid nanoparticle-mediated vectors are being explored to deliver multiple nucleic acid forms,including pDNA,mRNA,siRNA,and proteins.Herein,we discuss the broader applications of lipid nanoparticles,from protein replacement therapy to restoring the disease mechanism through nucleic acid delivery and gene editing,as well as multiple preclinical and clinical studies as a potential alternative to liver transplantation. 展开更多
关键词 Lipid nanoparticle Liver disorders Gene therapy Gene editing Nucleic acid delivery PDNA mRNA siRNA ASO Clinical trials
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