Literature DB >> 25830689

Gene therapy for inherited diseases of liver metabolism.

Pasquale Piccolo1, Nicola Brunetti-Pierri.   

Abstract

Gene therapy is entering the stage of initial clinical development to treat a growing number of inherited metabolic diseases. This review outlines the development of liver-directed gene therapy for diseases caused by deficiencies of enzymes that are primarily expressed in the liver and discusses the disorders that appear most promising for clinical translation.

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Year:  2015        PMID： 25830689     DOI： 10.1089/hum.2015.029

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   5.695


  4 in total

Review 1.  Metabolic Liver Disease: When to Suspect and How to Diagnose?

Authors:  Seema Alam; Vikrant Sood
Journal:  Indian J Pediatr       Date:  2016-04-29       Impact factor: 1.967

2.  In Silico Modeling of Liver Metabolism in a Human Disease Reveals a Key Enzyme for Histidine and Histamine Homeostasis.

Authors:  Roberto Pagliarini; Raffaele Castello; Francesco Napolitano; Roberta Borzone; Patrizia Annunziata; Giorgia Mandrile; Mario De Marchi; Nicola Brunetti-Pierri; Diego di Bernardo
Journal:  Cell Rep       Date:  2016-05-26       Impact factor: 9.423

3.  Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1.

Authors:  R Castello; R Borzone; S D'Aria; P Annunziata; P Piccolo; N Brunetti-Pierri
Journal:  Gene Ther       Date:  2015-12-24       Impact factor: 5.250

Review 4.  A primer to gene therapy: Progress, prospects, and problems.

Authors:  Hidde A Zittersteijn; Manuel A F V Gonçalves; Rob C Hoeben
Journal:  J Inherit Metab Dis       Date:  2020-07-20       Impact factor: 4.982
  4 in total

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