Lipid-nanoparticle-enabled nucleic acid therapeutics for liver disorders  

作　　者：Porkizhi Arjunan Durga Kathirvelu Gokulnath Mahalingam Ashish Kumar Goel Uday George Zacharaiah Alok Srivastava Srujan Marepally 

机构地区：[1]Center for Stem Cell Research(A Unit of inStem,Bengaluru),Christian Medical College Campus,Bagayam,Vellore 632002,Tamil Nadu,India [2]Manipal academy for higher education,Mangalore 576104,Karnataka,India [3]Department of Hematology,Christian Medical College&Hospital,Vellore 632004,Tamil Nadu,India [4]Department of Hepatology,Christian Medical College&Hospital,Vellore 632004,Tamil Nadu,India

出　　处：《Acta Pharmaceutica Sinica B》2024年第7期2885-2900,共16页药学学报（英文版）

基　　金：the Department of Biotechnology,India,for the financial support through grants,BT/PR25841/GET/119/162/2017,BT/PR40446/COV/140/5/2021。

摘　　要：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.

关 键 词：Lipid nanoparticle Liver disorders Gene therapy Gene editing Nucleic acid delivery PDNA mRNA siRNA ASO Clinical trials 

分 类 号：R575[医药卫生—消化系统]