PMID- 35859692
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220722
IS  - 2329-0501 (Print)
IS  - 2329-0501 (Electronic)
IS  - 2329-0501 (Linking)
VI  - 26
DP  - 2022 Sep 8
TI  - Emerging therapeutic potential of adeno-associated virus-mediated gene therapy in 
      liver fibrosis.
PG  - 191-206
LID - 10.1016/j.omtm.2022.06.009 [doi]
AB  - Liver fibrosis is a wound-healing response that results from various chronic 
      damages. If the causes of damage are not removed or effective treatments are not 
      given in a timely manner, it will progress to cirrhosis, even liver cancer. 
      Currently, there are no specific medical therapies for liver fibrosis. 
      Adeno-associated virus (AAV)-mediated gene therapy, one of the frontiers of 
      modern medicine, has gained more attention in many fields due to its high safety 
      profile, low immunogenicity, long-term efficacy in mediating gene expression, and 
      increasingly known tropism. Notably, increasing evidence suggests a promising 
      therapeutic potential for AAV-mediated gene therapy in different liver fibrosis 
      models, which helps to correct abnormally changed target genes in the process of 
      fibrosis and improve liver fibrosis at the molecular level. Moreover, the 
      addition of cell-specific promoters to the genome of recombinant AAV helps to 
      limit gene expression in specific cells, thereby producing better therapeutic 
      efficacy in liver fibrosis. However, animal models are considered to be powerless 
      predictive of tissue tropism, immunogenicity, and genotoxic risks in humans. 
      Thus, AAV-mediated gene therapy will face many challenges. This review 
      systemically summarizes the recent advances of AAV-mediated gene therapy in liver 
      fibrosis, especially focusing on cellular and molecular mechanisms of transferred 
      genes, and presents prospective challenges.
CI  - (c) 2022 The Authors.
FAU - Bu, Fang-Tian
AU  - Bu FT
AD  - Inflammation and Immune Mediated Diseases Laboratory of Anhui Province, Anhui 
      Institute of Innovative Drugs, School of Pharmacy, Anhui Medical University, 81 
      Mei Shan Road, Hefei, Anhui Province 230032, China.
AD  - Institute for Liver Diseases of Anhui Medical University, Hefei, China.
FAU - Jia, Peng-Cheng
AU  - Jia PC
AD  - Inflammation and Immune Mediated Diseases Laboratory of Anhui Province, Anhui 
      Institute of Innovative Drugs, School of Pharmacy, Anhui Medical University, 81 
      Mei Shan Road, Hefei, Anhui Province 230032, China.
AD  - Institute for Liver Diseases of Anhui Medical University, Hefei, China.
FAU - Zhu, Yan
AU  - Zhu Y
AD  - The First Affiliated Hospital of Anhui Medical University, Hefei, China.
FAU - Yang, Ya-Ru
AU  - Yang YR
AD  - The Second Affiliated Hospital of Anhui Medical University, Hefei, China.
FAU - Meng, Hong-Wu
AU  - Meng HW
AD  - Inflammation and Immune Mediated Diseases Laboratory of Anhui Province, Anhui 
      Institute of Innovative Drugs, School of Pharmacy, Anhui Medical University, 81 
      Mei Shan Road, Hefei, Anhui Province 230032, China.
AD  - Institute for Liver Diseases of Anhui Medical University, Hefei, China.
FAU - Bi, Yi-Hui
AU  - Bi YH
AD  - The Second Affiliated Hospital of Anhui Medical University, Hefei, China.
FAU - Huang, Cheng
AU  - Huang C
AD  - Inflammation and Immune Mediated Diseases Laboratory of Anhui Province, Anhui 
      Institute of Innovative Drugs, School of Pharmacy, Anhui Medical University, 81 
      Mei Shan Road, Hefei, Anhui Province 230032, China.
AD  - Institute for Liver Diseases of Anhui Medical University, Hefei, China.
FAU - Li, Jun
AU  - Li J
AD  - Inflammation and Immune Mediated Diseases Laboratory of Anhui Province, Anhui 
      Institute of Innovative Drugs, School of Pharmacy, Anhui Medical University, 81 
      Mei Shan Road, Hefei, Anhui Province 230032, China.
AD  - Institute for Liver Diseases of Anhui Medical University, Hefei, China.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20220622
PL  - United States
TA  - Mol Ther Methods Clin Dev
JT  - Molecular therapy. Methods & clinical development
JID - 101624857
PMC - PMC9271983
OTO - NOTNLM
OT  - AAV
OT  - HSCs
OT  - cellular mechanism
OT  - gene therapy
OT  - liver fibrosis
COIS- The authors declare no competing interests.
EDAT- 2022/07/22 06:00
MHDA- 2022/07/22 06:01
PMCR- 2022/06/22
CRDT- 2022/07/21 02:16
PHST- 2022/07/21 02:16 [entrez]
PHST- 2022/07/22 06:00 [pubmed]
PHST- 2022/07/22 06:01 [medline]
PHST- 2022/06/22 00:00 [pmc-release]
AID - S2329-0501(22)00088-2 [pii]
AID - 10.1016/j.omtm.2022.06.009 [doi]
PST - epublish
SO  - Mol Ther Methods Clin Dev. 2022 Jun 22;26:191-206. doi: 
      10.1016/j.omtm.2022.06.009. eCollection 2022 Sep 8.