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2015 ; 21
(13
): 3813-25
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New gene therapy strategies for hepatic fibrosis
#MMPMID25852266
Salazar-Montes AM
; Hernández-Ortega LD
; Lucano-Landeros MS
; Armendariz-Borunda J
World J Gastroenterol
2015[Apr]; 21
(13
): 3813-25
PMID25852266
show ga
The liver is the largest internal organ of the body, which may suffer acute or
chronic injury induced by many factors, leading to cirrhosis and hepatocarcinoma.
Cirrhosis is the irreversible end result of fibrous scarring and hepatocellular
regeneration, characterized by diffuse disorganization of the normal hepatic
structure, regenerative nodules and fibrotic tissue. Cirrhosis is associated with
a high co-morbidity and mortality without effective treatment, and much research
has been aimed at developing new therapeutic strategies to guarantee recovery.
Liver-based gene therapy has been used to downregulate specific genes, to block
the expression of deleterious genes, to delivery therapeutic genes, to prevent
allograft rejection and to augment liver regeneration. Viral and non-viral
vectors have been used, with viral vectors proving to be more efficient. This
review provides an overview of the main strategies used in liver-gene therapy
represented by non-viral vectors, viral vectors, novel administration methods
like hydrodynamic injection, hybrids of two viral vectors and blocking molecules,
with the hope of translating findings from the laboratory to the patient's
bed-side.
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