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Comparison of gene therapy strategies for treatment of liver diseases
Author(s) -
ROYCHOWDHURY NAMITA,
LU YANG,
JIANG JINLAN,
LEE SUNG W,
GUHA CHANDAN,
ROYCHOWDHURY JAYANTA
Publication year - 2004
Publication title -
journal of gastroenterology and hepatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.214
H-Index - 130
eISSN - 1440-1746
pISSN - 0815-9319
DOI - 10.1111/j.1440-1746.2004.03691.x
Subject(s) - genetic enhancement , gene , context (archaeology) , medicine , in vivo , computational biology , gene product , gene expression , nucleic acid , rna , recombinant dna , viral vector , bioinformatics , biology , cancer research , genetics , paleontology
Liver‐directed gene therapy can be aimed at replacing a missing gene product, overexpressing or ectopically expressing a gene product in the liver, generating proteins that are normally not produced in the liver (e.g. hormones, vaccines), down‐regulating specific gene expression and targeted repair of genetic mutations. A common critical requirement for achieving these goals is the availability of efficient methods for transferring DNA and RNA into target organ in vivo . Both recombinant viruses and non‐viral vectors are being explored for transferring nucleic acids to cells in vitro and in vivo . This review compares the characteristics of these vectors in the context of their potential application in liver‐directed gene therapy for various inherited or acquired disorders.