Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders.

ABSTRACT: The majority of monogenic liver diseases are autosomal recessive disorders, with few being sex-related or co-dominant. Although orthotopic liver transplantation (LT) is currently the sole therapeutic option for end-stage patients, such an invasive surgical approach is severely restricted by the lack of donors and post-transplant complications, mainly associated with life-long immunosuppressive regimens. Therefore, the last decade has witnessed efforts for innovative cellular or gene-based therapeutic strategies. Gene therapy is a promising approach for treatment of many hereditary disorders, such as monogenic inborn errors. The liver is an organ characterized by unique features, making it an attractive target for in vivo and ex vivo gene transfer. The current genetic approaches for heredit

SUBMITTER: Ghasemzad M 

PROVIDER: S-EPMC9404740 | biostudies-literature | 2022 Aug

REPOSITORIES: biostudies-literature