Hemophilia Gene Therapy: From Bench to Bed Side.

Hemophilia Gene Therapy: From Bench to Bed Side.
Curr Gene Ther. 2019 Sep 24;:
Authors: Guo XL, Chung TH, Qin Y, Zheng J, Zheng H, Sheng L, Wynn T, Chang LJ
Abstract
Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review will address the latest gene therapy research in hemophilia A and B including gene therapy tools, genetic strategies and animal models, and summarizes results of recent clinical trials. We will also discuss potential solutions to the current barriers in gene therapy for hemophilia.
PMID: 31549954 [PubMed - as supplied by publisher]

Source: Current Gene Therapy - September 23, 2019 Category: Genetics & Stem Cells Authors: Guo XL, Chung TH, Qin Y, Zheng J, Zheng H, Sheng L, Wynn T, Chang LJ Tags: Curr Gene Ther Source Type: research