Gene Therapy in Tyrosinemia: Potential and Pitfalls.

Abstract:

:In this chapter, we intend to review gene therapy concepts applied to the potential treatment of tyrosinemia for parents and pediatricians. Therefore, our main objective is to give general informations in a comprehensible manner. Considering the nature of tyrosinemia and the current state of technology, a particular focus will be put on strategies using viral delivery of DNA to the liver. In light of the recent development of the CRISPR technology and the revival of promises for previously unavailable therapeutical tools, the present chapter aims at presenting up to date facts and potential pitfalls towards an application for metabolic diseases, in particular tyrosinemia.

journal_name

Adv Exp Med Biol

authors

Carter S,Doyon Y

doi

10.1007/978-3-319-55780-9_21

subject

Has Abstract

pub_date

2017-01-01 00:00:00

pages

231-243

eissn

0065-2598

issn

2214-8019

journal_volume

959

pub_type

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